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3668 Study Matches

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Adrenoleukodystrophy National Registry Study

In this protocol, we will enroll pediatric, adolescent and adult patients diagnosed with adrenoleukodystrophy (ALD). These patients will include probands diagnosed by newborn screening and their relatives subsequently diagnosed, as well other patients who are diagnosed with ALD due to other presenting signs and symptoms and subsequently were confirmed to have ALD. We will ask consenting subjects to provide a medical history (with verification via medical records), to participate in a semi-annual health survey and provide consent to collect biospecimens. The overarching goal of this work is to engage with families affected by ALD and to assemble a resource of clinical, medical, and biological data that will allow of to better understand the natural history of ALD, and how this is affected by newborn screening. The initial focus will be on patients within Minnesota, but participation will be open to any family interested in the study, as this will be web-based. This registry and biobank, together with other research conducted in tandem, will possibly provide information describing the natural history of ALD and outcomes with interventions. It is anticipated that the data collected will further our understanding of the natural history of the disease, basic biology of adrenoleukodystrophy, diagnosis and outcomes. Ultimately, this research may lead to new avenues for early diagnosis and development of safer and more effective therapies for ALD.

Ashish Gupta
gupta461@umn.edu
All
Not specified
This study is NOT accepting healthy volunteers
NCT03789721
STUDY00003605
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Inclusion Criteria
• Age 0
•100
• ALD patients or family member meeting any of the following criteria:
• Any patient diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation).
• Known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene
• Participants living in the United States and territories Exclusion Criteria
• Patients diagnosed with ALD who lack the capacity to consent/assent AND do not have a designated legally authorized representative or guardian.
• Patients who have undergone BMT or other cellular therapy .
• Patients not fluent in English who are unable to consent in-person at the BMT Journey Clinic.
• Patients who are illiterate
• Patient determined by the PI or designee to be unlikely to complete required study components (due to language barriers, compliance issues, etc.)
Other: Medical Record Abstraction, Other: Biospecimen Sample Collection
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Registry, VLCFA, ABCD1, X-chromosome
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Masonic Cancer Center at University of Minnesota — Minneapolis, Minnesota Phil Lacher - (placher@umn.edu)

Randomized Phase II and Phase III Studies of Individualized Treatment for Nasopharyngeal Carcinoma Based on Biomarker Epstein Barr Virus (EBV) Deoxyribonucleic Acid (DNA)

Individualized Treatment in Treating Patients With Stage II-IVB Nasopharyngeal Cancer Based on EBV DNA

Katharine Price
All
18 years and over
Phase 2/3
This study is NOT accepting healthy volunteers
0000-100305-P01-RST
18-000158
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Inclusion Criteria:

  • Biopsy proven (from primary lesion and/or lymph nodes) diagnosis of cancer of the nasopharynx
  • Patients must have detectable pretreatment plasma EBV DNA, determined by the central lab prior to Step 2 registration
  • Stage II-IVB disease (American Joint Committee on Cancer [AJCC], 7th edition [ed.]) with no evidence of distant metastasis, based upon the following minimum diagnostic workup:
    • History/physical examination by a Medical Oncologist or Clinical Oncologist or Radiation Oncologist or Ear, Nose, Throat specialist (ENT), which must include an endoscopic evaluation, a complete list of current medications, and assessment of weight and weight loss in the past 6 months within 21 days prior to registration
    • Evaluation of tumor extent with magnetic resonance imaging (MRI) of the nasopharynx and neck within 28 days prior to registration; if MRI is medically contraindicated, obtain computed tomography (CT) scan with =< 3 mm contiguous slices with contrast and bone windows (to evaluate base of skull involvement); Note: If a treatment planning CT scan is used, it must be with =< 3 mm contiguous slices with contrast and be read by a radiologist
    • To rule out distant metastasis, patients must undergo the following imaging within 28 days prior to registration:
      • A CT scan with contrast of the chest, abdomen, and/or pelvis or a total body positron emission tomography (PET)/CT scan (non-contrast PET/CT is acceptable)
      • A bone scan only when there is suspicion of bone metastases (a PET/CT scan can substitute for the bone scan)
  • Zubrod performance status 0-1 within 21 days prior to registration
  • Absolute neutrophil count (ANC) >= 1,500 cells/mm^3
  • Platelets >= 100,000 cells/mm^3
  • Hemoglobin >= 8.0 g/dl (Note: the use of transfusion or other intervention to achieve hemoglobin [Hgb] >= 8.0 g/dl is acceptable)
  • Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 1.5 x institutional ULN
  • Alkaline phosphatase =< 1.5 x institutional ULN
  • Serum creatinine =< 1.5 mg/dl or calculated creatinine clearance (CC) >= 50 ml/min determined by 24-hour urine collection or estimated by Cockcroft-Gault formula
  • Negative serum pregnancy test within 14 days prior to registration for women of childbearing potential
  • Women of childbearing potential and male participants who are sexually active must agree to use a medically effective means of birth control throughout protocol treatment
  • Patient must provide study specific informed consent prior to study entry, including the mandatory pre-treatment plasma EBV DNA assay


Exclusion Criteria:

  • Prior invasive malignancy (except node negative, non-melanomatous skin cancer) unless disease free for a minimum of 1095 days (3 years) (for example, carcinoma in situ of the breast, oral cavity, or cervix are all permissible)
  • Prior systemic chemotherapy for the study cancer; note that prior chemotherapy for a different cancer is allowable; however, at least 6-weeks recovery is necessary if the last regimen included nitrosourea or mitomycin
  • Prior radiotherapy to the region of the study cancer that would result in overlap of radiation therapy fields
  • Patients with hearing loss assessed to be primarily sensorineural in nature, requiring a hearing aid, or intervention (i.e. interfering in a clinically significant way with activities of daily living); a conductive hearing loss from tumor-related otitis media is allowed
  • >= Grade 2 peripheral sensory neuropathy (CTCAE, v. 4.0)
  • Severe, active co-morbidity, defined as follows:
    • Major medical or psychiatric illness, which in the investigator's opinion would interfere with the completion of therapy and follow up or with full understanding of the risks and potential complications of the therapy
    • Unstable angina and/or uncontrolled congestive heart failure
    • Myocardial infarction within the last 6 months
    • Acute bacterial or fungal infection requiring intravenous antibiotics at the time of registration; note that patients switched from IV antibiotics and currently on oral antibiotics whose infection is assessed to be adequately treated or controlled are eligible
    • Chronic obstructive pulmonary disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy within 30 days prior to registration
    • Acquired immune deficiency syndrome (AIDS) based upon current Centers for Disease Control and Prevention (CDC) definition; note, however, that human immunodeficiency virus (HIV) testing is not required for entry into this protocol
  • Pregnancy or women of childbearing potential and men who are sexually active and not willing/able to use medically acceptable forms of contraception
  • Prior allergic reaction to the study drug(s) involved in this protocol
  • Patients with undetectable pre-treatment plasma EBV DNA

Drug, Radiation, Administration of antineoplastic agent, Drug therapy, Epstein-Barr virus serologic test, Radiation therapy procedure or service, Radiation oncology AND/OR radiotherapy
Cancer, Head and neck cancer, Nasal and paranasal tumors, Nasal cancer, Nasopharyngeal carcinoma, Throat cancer
1,2-Diaminocyclohexaneplatinum II citrate, Cancer treatment, Chemotherapy, Digestive system, Fluorouracil [USAN:USP:INN:BAN:JAN], Gemcitabine [USAN:INN:BAN], Infinnium, Malignant tumor of nasopharynx, Medical Oncology, Radiation therapy, Respiratory system, cisplatin, fluorouracil, gemcitabine, paclitaxel
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Mayo Clinic — Rochester, MN

Ventricular and Vascular Function Abnormalities in Patients with Coarctation of Aorta: The Role of Exercise Echocardiography

A Study to Analyze if Exercise Echocardiography Can Identify Ventricular and Vascular Function Abnormalities That are Undetectable at Rest for Patients With Coarctation of Aorta (COA)

Alexander Egbe
All
18 years and over
Not Applicable
This study is NOT accepting healthy volunteers
0000-120334-H01-RST
17-010089
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Inclusion Criteria:

  • Age ≥ 18 years old.
  • History of COA.


Exclusion Criteria:

  • Patients under 18 years old.

 

Other, Radiation, Exercise stress echocardiography
Coarctation of the aorta
Cardiovascular system, Coarctation of aorta, Exercise stress test
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Mayo Clinic — Rochester, MN

A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy (METRE-PL)

Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Patients With PL

A.J. Vinaya Simha
All
18 years and over
Phase 3
This study is NOT accepting healthy volunteers
2022-308741-P01-RST
22-007213
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Inclusion Criteria:

  • Diagnosis of Partial Lipodystrophy.


Exclusion Criteria:

  • Previous treatment with metreleptin.

Eligibility last updated 7/7/22. Questions regarding updates should be directed to the study team contact.

 

Drug, Other
Familial partial lipodystrophy, metreleptin
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Mayo Clinic — Rochester, MN

An Open-Label, Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant

A Study of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant

William Hogan
All
18 years and over
Not Applicable
This study is NOT accepting healthy volunteers
0000-122186-P01-RST
19-004906
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Inclusion Criteria:

  • Male or female, 12 years of age or older.
  • Have undergone an allo-HSCT from any donor source using bone marrow, peripheral blood stem cells, or cord blood for hematologic malignancies. Recipients of nonmyeloablative and myeloablative conditioning regimens are eligible.
  • Clinically suspected all grades acute or chronic GVHD as per Minnesota-Center for International Blood and Marrow Transplant Research (MN-CIBMTR) criteria, that is refractory or intolerant to corticosteroids, occurring after allo-HSCT with any conditioning regimen and any anti-GVHD prophylactic program. Clinical suspicion of GVHD by the treating physician is also sufficient. 
  • Evidence of myeloid engraftment (e.g., absolute neutrophil count ≥ 1.0 × 10^9/L for 3 consecutive days if ablative therapy was previously used). Use of growth factor supplementation is allowed. 
  • Evidence of platelet engraftment (i.e., platelets ≥ 20 × 10^9/L). 
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 3. 
  • Be willing to avoid pregnancy or fathering children based on 1 of the following criteria: 
    • Women of non-childbearing potential (i.e., surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea);
    • Woman of childbearing potential who has a negative serum pregnancy test at screening and who agrees to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the patient and their understanding confirmed;
    • Man who agrees to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the patient and their understanding confirmed.
  • Able to provide written informed consent and/or assent from the patient, parent, or guardian.

 


Exclusion Criteria:
 

  • Eligible for an existing and actively enrolling Incyte sponsored clinical trial for ruxolitinib for the treatment of GVHD. 
  • Patients or legal guardians unable to review and sign informed consent form.
  • Females who are pregnant or breastfeeding, and males and females who cannot comply with requirements to avoid fathering a child or becoming pregnant. 
  • Patients with inadequate liver function (alanine aminotransferase above 4 × upper limit of normal (ULN) or direct bilirubin 4 × ULN and the laboratory abnormalities are considered to be due to underlying liver dysfunction) unless attributed to GVHD. 
  • Patients with end stage renal function (creatinine clearance (CrCl) < 15 mL/min or glomerular filtration rate < 15 mL/min), regardless of whether hemodialysis is required. 
  • Any underlying or current medical or psychiatric condition that, in the opinion of the treating physician, would place the patient at an unacceptable risk if he or she were to participate in the program. 
  • Previous allergic reactions to Janus kinase (JAK) inhibitors or excipients. 
  • Patients who are currently taking any anticancer therapy (e.g., chemotherapy, radiation therapy, surgery, immunotherapy, biologic therapy, hormonal therapy, or tumor embolization). 
  • Patients taking any secondary GVHD therapy due to insufficient response/progression on program treatment including, but not limited to, ibrutinib, filgotinib, and other off-label medications.
  • Concomitant use of any JAK inhibitor.
  • Initiating therapy with any investigational medication. 
  • Presence of an active uncontrolled infection. An active uncontrolled infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs, or radiographic findings attributable to infection. Persisting fever without signs or symptoms will not be interpreted as an active uncontrolled infection. 
  • Known HIV infection. 
  • Active hepatitis B virus (HBV) or hepatitis C virus infection that requires treatment or at risk for HBV reactivation. At risk for HBV reactivation is defined as hepatitis B surface antigen positive or anti-hepatitis B core antibody positive. Previous test results obtained as part of standard of care before allo-HSCT that confirm a patient is immune and not at risk for reactivation (i.e., hepatitis B surface antigen negative, surface antibody positive) may be used for purposes of eligibility.
Drug, Administration of antineoplastic agent, Allogeneic bone marrow transplantation, Drug therapy
Cancer, Graft versus host disease, Leukemia, Lymphoma, Pediatric white blood cell disorders
Allogeneic stem cell transplant, Bone marrow transplant, Cancer treatment, Chemotherapy, Graft versus host disease, Hematologic neoplasm, Hematopoietic system, Malignant neoplastic disease, Medical Oncology, Ruxolitinib [USAN:INN], ruxolitinib
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Mayo Clinic — Rochester, MN

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

Ashish Gupta
gupta461@umn.edu
All
2 Years to 50 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293185
STUDY00006923
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Inclusion Criteria:

• Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
• Be ≥2 and ≤50 years of age at time of consent.
• Weigh a minimum of 6 kg.
• Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age).
• Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
• In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent.
• Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
• Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
• Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
Exclusion Criteria:

• Subjects for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available.
• Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for subjects ≤ 16 years of age (e.g. TCD velocity >200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
• Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis.
• Clinically significant, active bacterial, viral, fungal, or parasitic infection
• Advanced liver disease, such as 1. clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy) 2. liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
• Inadequate bone marrow function, as defined by an absolute neutrophil count of <1×10^9/L (<0.5×10^9/L for subjects on hydroxyurea treatment) or a platelet count <100×10^9/L.
• Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
• Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
• Unable to receive pRBC transfusion.
• Prior receipt of an allogeneic transplant.
• Prior receipt of gene therapy.
• Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
• Immediate family member with a known or suspected Familial Cancer Syndrome.
• Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion.
• Any other condition that would render the subject ineligible for HSCT.
• Participation in another clinical study with an investigational drug within 30 days of screening.
• Presence of a chromosomal abnormality or genetic mutation that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
• Presence of genetic mutations that result in the inactivation of 2 or more α-globin genes
Genetic: bb1111
Sickle Cell Disease
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University of Minnesota — Minneapolis, Minnesota

ACCL2031, A Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine (IND #149832) for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors (ACCL2031)

A Study to See if Memantine Protects the Brain During Radiation Therapy Treatment for a Brain Tumor

Nadia Laack
All
4 years to 17 years old
Phase 3
This study is NOT accepting healthy volunteers
2020-302527-P01-RST
20-010883
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Inclusion Criteria:


- >= 4 and < 18 years at time of study entry

- Patients must weigh 15 kg or greater at time of study entry

- Newly diagnosed or recurrent primary brain tumors that have not received prior cranial
radiotherapy

- Planned focal, cranial or craniospinal radiation treatment for a primary brain tumor

- The patient must have receptive and expressive language skills in English, French or
Spanish since the neurocognitive function and quality of life (QOL) assessment
instruments are available in these languages only

- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:

- Age: 4 to < 6 years; Maximum serum creatinine (mg/dL): 0.8 male; 0.8 female

- Age: 6 to < 10 years; Maximum serum creatinine (mg/dL): 1 male; 1 female

- Age: 10 to < 13 years; Maximum serum creatinine (mg/dL): 1.2 male; 1.2 female

- Age: 13 to < 16 years; Maximum serum creatinine (mg/dL): 1.5 male; 1.4 female

- Age: >= 16 years; Maximum serum creatinine (mg/dL): 1.7 male; 1.4 female

- Total bilirubin =< 1.5 x upper limit of normal (ULN) for age

- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L

- Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the
value of 45 U/L

- The patient must be able to undergo magnetic resonance imaging

- All patients and/or their parents or legal guardians must sign a written informed
consent

- All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met


Exclusion Criteria:


- Life expectancy of less than 18 months

- Pre-existing conditions:

- Any contraindication or allergy to memantine

- Intractable seizures while on adequate anticonvulsant therapy, defined as more
than one seizure per month for the past 2 months or since initiating
anticonvulsant therapy

- Co-morbid systemic illnesses, psychiatric conditions, social situations, or other
severe concurrent disease which, in the judgment of the investigator, would make
the patient inappropriate for entry into this study or interfere significantly
with the proper assessment of safety and toxicity of the prescribed regimens or
would limit compliance with the study requirements

- Patients with a motor, visual, or auditory condition that precludes computerized
neurocognitive assessments are not eligible to participate

- Patients with any medical condition or taking medications that lead to
alterations of urine pH towards the alkaline condition (e.g., renal tubular
acidosis, carbonic anhydrase inhibitors, sodium bicarbonate)

- Personal history of prior cranial or craniospinal radiotherapy is not allowed

- Note: Prior anti-cancer therapy including surgery, chemotherapy, targeted agents
are allowed as per standard of care clinical treatment guidelines

- Female patients who are pregnant are excluded since fetal toxicities and teratogenic
effects have been noted for the study drug. A pregnancy test is required for female
patients of childbearing potential

- Lactating females who plan to breastfeed their infants

- Sexually active patients of reproductive potential who do not agree to use an
effective contraceptive method for the duration of their study participation

Drug, Procedure/Surgery, Other
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Mayo Clinic — Rochester, MN

Evaluation of the Single DNA Molecule Optical Imaging (Bionano) Analysis as a Tool for Facioscapulohumeral Muscular Dystrophy (FSHD) Diagnosis (FSHD)

A Study to Evaluate Single DNA Molecule Optical Imaging to Diagnose Facioscapulohumeral Muscular Dystrophy (FSHD)

Zhiyv Niu
All
18 years and over
This study is NOT accepting healthy volunteers
0000-123106-H01-RST
20-000364
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Inclusion Criteria:

  • Patients with confirmed Facioscapulohumeral muscular dystrophy (FSHD) diagnosis with conventional genetic testing.
  • Patients with clinically suspected FSHD but with inconclusive conventional genetic testing result or negative conventional genetic testing for FSHD and FSHD mimickers.
  • 20 De-identified reference samples at Genomic Laboratories to test specimen types.


Exclusion Criteria:

  • Patients with genetically characterized muscle disease different than FSHD.   

 

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Mayo Clinic — Rochester, MN

An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Pegcetacoplan in Patients with Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT) (Sobi)

A Study to Evaluate the Pharmacokinetics (PK), Safety and Tolerability of Pegcetacoplan in Patients With TA-TMA After Hematopoietic Stem Cell Transplantation (HSCT)

Hassan Alkhateeb
All
18 years and over
Phase 2
This study is NOT accepting healthy volunteers
2022-307190-P01-RST
22-001210
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Inclusion Criteria:

  • Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
  • Received allogeneic HSCT from a related or unrelated, human leukocyte antigen-matched or mismatched donor. Patients having received any of the following stem cell sources are eligible: granulocyte colony stimulating factor mobilized peripheral blood stem cells, bone marrow, umbilical cord blood.
  • Diagnosis of TA-TMA established by histologic evidence of microangiopathy in any biopsied organ OR, as per the laboratory markers below, indicating TMA:
    • De novo or progressing thrombocytopenia (platelet count < 50 x 10^9/L or > 50 % decrease in platelet count from the highest value achieved after transplantation); AND
    • Elevated LDH (> 1.5 x ULN); AND
    • At least 1 additional laboratory criteria among the following:
    • Schistocytes on the peripheral blood smear (≥ 2 per hpf); OR
    • De novo anemia (hemoglobin < LLN or anemia requiring PRBC transfusion support as per local institutional standard); OR
    • Proteinuria (rUPCR ≥ 2 mg/mg); OR
    • Elevated plasma concentration of sC5b-9 above ULN.
  • Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
  • Have at least 1 sign/symptom of organ dysfunction:
    • Kidney: doubling of serum creatinine compared with pre-HSCT level or patient receiving renal replacement therapy or proteinuria ≥ 30 mg/dL AND rUPCR ≥ 2 mg/mg;
    • Lungs: hypoxemia or any need for noninvasive or invasive positive pressure ventilation;
    • Cardiovascular: pulmonary hypertension diagnosed by a cardiologist using cardiac catheterization, or pulmonary hypertension criteria on echocardiography or arterial hypertension, defined by systolic blood pressure (BP) ≥ 140 mmHg and/or diastolic BP ≥ 90 mmHg at baseline or hypertension requiring > 2 medications (excluding diuretics);
    • Serositis: clinically significant pleural effusion or pericardial effusion requiring surgical therapy (e.g., pericardiocentesis/ thoracocentesis);
    • CNS: seizures attributable to posterior reversible encephalopathy syndrome;
    • GI tract: presence of biopsy-proven GI TA-TMA. Patients with GI bleeding (hematemesis or hematochezia) will be excluded.
  • Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
    • Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
  • Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
    • Avoid fathering a child;
    • Use protocol-defined methods of contraception;
    • Refrain from donating sperm.
  • Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.


Exclusion Criteria:

  • Positive direct Coombs test.
  • Known familial or acquired ADAMTS13 deficiency.
  • Known Shiga toxin‐related hemolytic uremic syndrome.
  • Known bone marrow or graft failure.
  • Diagnosis of disseminated intravascular coagulation.
  • Diagnosis of VOD.
  • Active GI bleeding (hematemesis or hematochezia) at baseline.
  • Body weight < 30 kg and > 100 kg.
  • Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis.
  • Previously or currently treated with a complement inhibitor (approved or investigational).
  • Pregnancy or breastfeeding.
  • Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record.
  • Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record.
  • Chronic inactive hepatitis B virus with viral loads > 1000 IU/mL (> 5000 copies/mL) at screening or documented in pre-HSCT medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
  • Inability to cooperate with study procedures or any condition that, in the opinion of the investigator, could increase the patient’s risk by participating in the study or confound the outcome of the study.

Eligibility last updated 2/2/22. Questions regarding updates should be directed to the study team contact.

Drug
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A Prospective, Multicenter, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of PMX Cartridge in Addition to Standard Medical Care for Patients With Endotoxemic Septic Shock (TIGRIS)

Safety and Efficacy of Polymyxin B Hemoperfusion (PMX) for Endotoxemic Septic Shock in a Randomized, Open-Label Study

Kianoush Banaei Kashani
All
18 years and over
Not Applicable
This study is NOT accepting healthy volunteers
2022-309695-P01-RST
22-010446
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Inclusion Criteria:


1. Age ≥18 years of age.

2. Hypotension requiring vasopressor support: Requirement for at least one of the
vasopressors listed below, at the dose shown below, for at least 2 continuous hours
and no more than 30 hours:

  • Norepinephrine > 0.05mcg/kg/min.;
  • Dopamine > 10 mcg/kg/min.;
  • Phenylephrine > 0.4 mcg/kg/min.;
  • Epinephrine > 0.05 mcg/kg/min.;
  • Vasopressin > 0.03 units/min.;
  • Vasopressin (any dose) in combination with another vasopressor listed above.

3. The subject must have received intravenous fluid resuscitation of a minimum of 30mL/kg
administered within 24 hours of eligibility.

4. Documented or suspected infection defined as definitive or empiric intravenous
antibiotic administration.

5. The subject must have a screening multi-organ dysfunction score (MODS) > 9 OR a
sequential organ failure assessment (SOFA) >11, in the event a complete MODS cannot be
obtained due to missing measurements.

6. Endotoxin Activity Assay between ≥ 0.60 to <0.90 EA units.

7. Evidence of at least 1 of the following criteria for new onset organ dysfunction that
is considered to be due to the acute illness:

  • Requirement for positive pressure ventilation via an endotracheal tube or tracheostomy tube;
  • Thrombocytopenia defined as acute onset of platelet count < 150,000µ/L or a reduction of 50% from prior known levels;
  • Acute oliguria defined as urine output < 0.5mL/kg/hr for at least 6 hours despite adequate fluid resuscitation.


Exclusion Criteria:


1. Inability to obtain an informed consent from the subject, family member or an
authorized surrogate.

2. Lack of commitment for full medical support.

3. Inability to achieve or maintain a minimum mean arterial pressure (MAP) of ≥ 65mmHg
despite vasopressor therapy and fluid resuscitation.

4. Subject has end-stage renal disease and requires chronic dialysis.

5. There is clinical support for non-septic shock such as:

  • Acute pulmonary embolus;
  • Transfusion reaction;
  • Severe congestive heart failure (e.g., NYHA Class IV, ejection fraction < 35%).

6. Subject has had chest compressions as part of CPR during this hospitalization without
immediate return to communicative state.

7. Subject has had an acute myocardial infarction (AMI) within the past 4 weeks.

8. Subject has uncontrolled hemorrhage (acute blood loss requiring > 3 UPC in the past 24
hours).

9. Major trauma within 36 hours of screening.

10. Subject has severe granulocytopenia (leukocyte count less than 500 cells/mm3) or
severe thrombocytopenia (platelet count less than 30,000 cells/mm^3).

11. HIV infection in association with a last known or suspected CD4 count of < 50/mm^3.

12. Subject's baseline state is non-communicative.

13. Subject has sustained extensive third-degree burns within the past 7 days.

14. Body weight < 35 kg (77 pounds).

15. Known hypersensitivity to Polymyxin B.

16. Subject has known sensitivity or allergy to heparin or has a history of heparin
associated thrombocytopenia (H.I.T.).

17. Subject is currently enrolled in an investigational drug or device trial.

18. Subject has been previously enrolled in the current trial.

19. Any other condition, that in the opinion of the investigator, would preclude the
subject from being a suitable candidate for enrollment, such as end-stage chronic
illness (e.g., lack of source control and bowel necrosis) with no reasonable expectation
of survival to hospital discharge.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 10/3/22. Questions regarding updates should be directed to the study team contact.

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Best Available Therapy Versus Autologous Hematopoetic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) (BEAT-MS)

The overall objective of this study is to compare the efficacy, safety, immunologic effects, and cost-effectiveness of myeloablative and immunoablative therapy followed by autologous hematopoietic stem cell transplant (AHSCT) versus best available therapy (BAT) over 72 months in participants with relapsing MS and continued MS disease activity despite treatment with DMTs. The primary efficacy objective is to compare MS relapse-free survival, analyzed as time until MS relapse or death from any cause.

Adam Carpenter
carpe004@umn.edu
All
18 Years to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04047628
STUDY00007288
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Inclusion Criteria:
1. Age 18 to 55 years, inclusive, at the time of the screening Visit -2. 2. Diagnosis of MS according to the 2017 McDonald Criteria139. 3. EDSS ≤ 6.0 at the time of randomization (Day 0). 4. T2 abnormalities on brain MRI that fulfill the 2017 McDonald MRI criteria for dissemination in space139. A detailed MRI report or MRI images must be available for review by the site neurology investigator. 5. Highly active treatment-resistant relapsing MS, defined as ≥ 2 episodes of disease activity in the 36 months prior to the screening visit (Visit -2). The two disease activity episodes will be a clinical MS relapse or MRI evidence of MS disease activity and must meet all the criteria described below: 1. At least one episode of disease activity must occur following ≥ 1 month of treatment with one of the following: (i) an oral DMT approved by the FDA for the treatment of relapsing MS, or (ii) a monoclonal antibody approved by the FDA for the treatment of relapsing MS, or (iii) rituximab. Qualifying DMTs include: dimethyl fumarate, diroximel fumarate, monomethyl fumarate, teriflunomide, cladribine, daclizumab, ponesimod, siponimod, ozanimod, fingolimod, rituximab, ocrelizumab, natalizumab, alemtuzumab, ublituximab, and ofatumumab, and 2. At least one episode of disease activity must have occurred within the 12 months prior to the screening visit (Visit -2), and 3. At least one episode of disease activity must be a clinical MS relapse (see item c.i. below). The other episode(s) must occur at least one month before or after the onset of the clinical MS relapse, and must be either another clinical MS relapse or MRI evidence of disease activity (see item c.ii. below): i. Clinical MS relapse must be confirmed by a neurologist's assessment and documented contemporaneously in the medical record. If the clinical MS relapse is not documented in the medical record, it must be approved by the study adjudication committee (see Section 3.5), and ii. MRI evidence of disease activity must include ≥ 1 unique active lesion on one or more brain or spinal cord MRIs. Detailed MRI reports or MRI images must be available for review by the site neurology investigator. A unique active lesion is defined as either of the following: 1. A gadolinium-enhancing lesion, or 2. A new non-enhancing T2 lesion compared to a reference scan obtained not more than 36 months prior to the screening visit (Visit -2). 6. Candidacy for treatment with at least one of the following high efficacy BAT DMTs: cladribine, natalizumab, alemtuzumab, ocrelizumab, ofatumumab, ublituximab and rituximab. Candidacy for treatment for each BAT DMT is defined as meeting all of the following: 1. No prior disease activity episode, as defined in Inclusion Criterion #5, with the candidate BAT DMT, and 2. No contraindication to the candidate BAT DMT, and 3. No treatment with the candidate BAT DMT in the 12 months prior to screening. 7. Completion of COVID-19 vaccination series, according to the current Centers for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) recommendations, ≥ 14 days prior to randomization (Day 0). 8. Positive for VZV antibodies, or completion of at least one dose of the varicella zoster glycoprotein E (gE) Shingrix vaccine at least 4 weeks prior to randomization (Day 0). 9. Insurance approval for MS treatment with at least one candidate BAT DMT (see Inclusion Criterion #6). 10. Ability to comply with study procedures and provide informed consent, in the opinion of the investigator. 11. Females of childbearing potential (defined in Section 5.4.3.1) and males with female partners of childbearing potential are required to adhere to the contraception provisions of Section 5.4.3.1. 12. For participants who use medicinal or recreational marijuana, willingness to substitute MARINOL® if randomized to AHSCT (Section 5.4.2.6).
Exclusion Criteria:
1. Diagnosis of primary progressive MS according to the 2017 McDonald criteria. 2. History of neuromyelitis optica spectrum disorder or MOG antibody disease. 3. Prior treatment with an investigational agent within 3 months or 5 half-lives, whichever is longer. Agents authorized by the FDA for prevention or treatment of COVID-19 are not considered investigational. 4. Either of the following within one month prior to randomization (Day 0): 1. Onset of acute MS relapse, or 2. Treatment with intravenous methylprednisolone 1000 mg/day for 3 days or equivalent. 5. Initiation of any BAT DMT (see Section 5.2.1) between Visit -2 and randomization (Day 0). 6. Brain MRI or cerebrospinal fluid (CSF) examination indicating a diagnosis of progressive multifocal leukoencephalopathy (PML). 7. History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS). 8. Presence of unexplained cytopenia, polycythemia, thrombocythemia or leukocytosis. 9. History of sickle cell anemia or other hemoglobinopathy. 10. Evidence of past or current hepatitis B or hepatitis C infection, including treated hepatitis B or hepatitis C. Hepatitis B surface antibody following hepatitis B immunization is not considered to be evidence of past infection. 11. Presence or history of mild to severe cirrhosis. 12. Hepatic disease with the presence of either of the following: 1. Total bilirubin ≥ 1.5 times the upper limit of normal (ULN) or total bilirubin ≥ 3.0 times the ULN in the presence of Gilbert's syndrome, or 2. Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥ 2.0 times the ULN. 13. Positive COVID-19 PCR test, or alternative nucleic acid amplification test (NAAT) per institutional standards, within 14 days prior to randomization (Day 0). 14. Evidence of HIV infection. 15. Positive QuantiFERON
•TB Gold,TB Gold Plus, or T-SPOT®.TB test results. PPD tuberculin test may be substituted for QuantiFERON
•TB Gold, TB Gold Plus, or T-SPOT®.TB test. 16. Active viral, bacterial, endoparasitic, or opportunistic infections. 17. Active invasive fungal infection. 18. Hospitalization for treatment of infections or parenteral (IV or IM) antibacterials, antivirals, antifungals, or antiparasitic agents within the 30 days prior to randomization (Day 0) unless clearance is obtained from an Infectious Disease specialist. 19. Receipt of live or live-attenuated vaccines within 6 weeks of randomization (Day 0). 20. Presence or history of clinically significant cardiac disease including: a. Arrhythmia requiring treatment with any antiarrhythmia therapy, with the exception of low dose beta blocker for intermittent premature ventricular contractions. b. Coronary artery disease with a documented diagnosis of either: i. Chronic exertional angina, or ii. Signs or symptoms of congestive heart failure. c. Evidence of heart valve disease, including any of the following: i. Moderate to severe valve stenosis or insufficiency, or ii. Symptomatic mitral valve prolapse, or iii. Presence of prosthetic mitral or aortic valve. 21. Left ventricular ejection fraction (LVEF) < 50%. 22. Impaired renal function defined as eGFR < 60 mL/min/1.73 m2, according to the CKD-EPI formula144. 23. Forced expiratory volume in one second (FEV1) < 70% predicted (no bronchodilator). 24. Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected for Hgb) < 70% predicted. 25. Poorly controlled diabetes mellitus, defined as HbA1c > 8%. 26. History of malignancy, except adequately treated localized basal cell or squamous skin cancer, or carcinoma in situ of the cervix. Malignancies for which the participant is judged to be cured will be considered on an individual basis by the study adjudication committee (see Section 3.5). 27. Presence or history of any moderate to severe rheumatologic autoimmune disease requiring treatment, including but not limited to the following: systemic lupus erythematous, systemic sclerosis, rheumatoid arthritis, Sjogren's syndrome, polymyositis, dermatomyositis, mixed connective tissue disease, polymyalgia rheumatica, polychondritis, sarcoidosis, vasculitis syndromes, or unspecified collagen vascular disease. 28. Presence of active peptic ulcer disease, defined as endoscopic or radiologic diagnosis of gastric or duodenal ulcer. 29. Prior history of AHSCT. 30. Prior history of solid organ transplantation. 31. Positive pregnancy test or breastfeeding. 32. Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy. 33. Psychiatric illness, mental deficiency, or cognitive dysfunction severe enough to interfere with compliance or informed consent. 34. History of hypersensitivity to rabbit or Escherichia coli-derived proteins. 35. Any metallic material or electronic device in the body, or other condition that precludes the participant from undergoing MRI with gadolinium administration, as determined by the site radiologist. 36. Presence or history of ischemic cerebrovascular disorders, including but not limited to transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage. 37. Presence or history of other neurological disorders, including but not limited to CNS or spinal cord tumor; metabolic or infectious cause of myelopathy; genetically-inherited progressive CNS disorder; CNS sarcoidosis; or systemic autoimmune disorders potentially causing progressive neurologic disease or affecting ability to perform the study assessments. 38. Presence of any medical comorbidity that the investigator determines will significantly increase the risk of treatment mortality. 39. Presence of any other concomitant medical condition that the investigator deems incompatible with trial participation.
Procedure: Autologous Hematopoietic Stem Cell Transplantation, Biological: Best Available Therapy (BAT)
Relapsing Multiple Sclerosis, Relapsing Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis
Treatment-Resistant Relapsing Multiple Sclerosis (MS), Autologous Hematopoietic Stem Cell Transplantation (AHSCT), Autologous Peripheral Blood Stem Cells (PBMCs) Graft, Best Available Therapy (BAT), Disease-Modifying Therapy (DMT), BAT DMT
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University of Minnesota Multiple Sclerosis Center — Minneapolis, Minnesota Emily Harper - (harpe442@umn.edu)

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

To determine the incidence of engraftment (defined as achieving donor derived neutrophil count >500/uL by day 42) in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.

Christen Ebens
ebens012@umn.edu
All
up to 3 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT00357565
0511M77206
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Inclusion Criteria:

• Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
• 1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg
• 2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg
• 3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg
• Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
• Acute myeloid leukemia: high risk CR1 as evidenced by:
• High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
• Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10% by a representative bone marrow aspirate morphology.
• Persistent or rising minimal residual disease (MRD) after standard chemotherapy regimens: Patients with evidence of minimal residual disease at the completion of therapy or evidence of rising MRD while on therapy. MRD will be defined by either flow cytometry (>0.1% residual cells in the blast gate with immune phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or conventional cytogenetics (g-banding).
• New Leukemia Subtypes: A major effort in the field of pediatric hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new high risk features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
• Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
• Renal: glomerial filtration rate > 60ml/min/1.73m^2
• Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,
• Pulmonary function: oxygen saturation >92%
• Cardiac: left ventricular ejection fraction > 45%.
• Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Exclusion Criteria:

• Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
• History of HIV infection or known positive serology
• Myeloablative transplant within the last 6 months.
• Evidence of active extramedullary disease (including central nervous system leukemia).
Biological: filgrastim, Drug: busulfan, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: melphalan, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantation
Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Previously Treated Myelodysplastic Syndrome, Secondary Myelodysplastic Syndrome, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Excess Blasts, Refractory Anemia, De Novo Myelodysplastic Syndrome, Childhood Myelodysplastic Syndrome
MDS, AML
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Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota Christen Ebens, MD - (ebens012@umn.edu)

A Phase 2 Randomized, Controlled, Dose-titration, Open-Label Study Evaluating the Safety and Efficacy of BIV201 in Addition to Standard of Care Compared to Standard of Care to Reduce the Recurrence of Ascites and Complications in Patients with Refractory Ascites Secondary to Decompensated Liver Cirrhosis

A Study Evaluating the Safety and Effectiveness of BIV201 and Standard of Care Compared to Standard of Care to Reduce the Recurrence of Ascites and Complications in Patients with Refractory Ascites Secondary to Decompensated Liver Cirrhosis

Douglas Simonetto
All
18 years to 75 years old
Phase 2
This study is NOT accepting healthy volunteers
2020-303189-P01-RST
20-013403
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Inclusion Criteria:

  • Informed consent, or consent via legally authorized representative, if institutionally required for subjects with decompensated liver cirrhosis with current HE Grade 1 or 2, prior to any study-related procedures.
  • Male or female patients age ≥ 18 years old.
  • Cirrhosis of the liver.
  • Patient has diuretic-resistant ascites, intractable ascites, or in the opinion of the Investigator, is unsuitable for treatment with an effective dose of diuretics for other reasons.
  • Patients must have required in the 60-day period from the last LVP before consent, between 3 and 9 LVPs, including the last LVP on or before the day of consent.
  • Dates for all LVPs occurring within 90 days prior to consent have been recorded. The volume of ascites removed at each of the LVPs must also have been recorded for the 90 days period prior to the last LVPs before consent.
  • Serum creatinine (SCr) ≤ 2.00 mg/dL determined prior to randomization with value at randomization being less than 1.5 fold higher than value obtained at the screening visit.
  • Women of child-bearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must be neither pregnant nor lactating and must agree to use adequate birth control or be abstinent for the duration of the study.
  • If patient is treated with beta blockers, dose has been stable for at least 30 days prior to randomization and may be maintained on that dose for the trial duration.
  • If patient is treated with diuretics, patient has been on a stable daily dose for at least 10 days prior to consent.
  • Willing and able to comply with trial instructions.


Exclusion Criteria:

  • Ascites with causes other than cirrhosis; such as cardiac or nephrogenic ascites or malignant ascites due to peritoneal carcinomatosis.
  • Urinary sodium excretion > 100 mmol/day between day of consent and randomization.
  • Total bilirubin > 5 mg/dL.
  • Blood clotting International normalized ratio (INR) > 2.5.
  • Platelet count ≤ 100,000/µL for the first eight patients enrolled. Eligibility of patients with a platelet count > 65,000 but < 100,000/µL should be confirmed with the Medical Monitor.
  • Current or recent (within 3 months of consent) renal dialysis.
  • Current or recent (within 1 month of consent) hepatic encephalopathy Grade 3 or 4 (WestHaven criteria).
  • Superimposed acute liver failure/injury due to factors including acute alcoholic hepatitis, acute viral hepatitis, drugs, medications (e.g., acetaminophen), or other toxins (e.g., mushroom [Amanita] poisoning).
  • History or presence of hepatic hydrothorax that has required a thoracentesis in the 7 days prior to randomization, or anticipated to require one within 7 days post-randomization, known pulmonary hypertension or a history of hepatopulmonary syndrome.
  • Current or recent treatment (within 7 days of randomization) with octreotide, midodrine, vasopressin, dopamine or other vasopressors.
  • Treatment with ACE inhibitors or ARBs 30 days prior to randomization.
  • Current or recent (in the previous 60 days from consent) episode of respiratory failure requiring positive airway pressure (PAP) devices or intubation.
  • Sepsis episode in the previous 28 days from consent.
  • Episode of SBP within 14 days prior to consent. If a patient develops SBP in the pretreatment period randomization cannot occur for a minimum of 10 days and documented resolution of SBP.
  • SBP at baseline visit (LVP -1).
  • Episode of gastrointestinal hemorrhage (non-variceal) within 28 days prior to consent.
  • Episode of bleeding esophageal varices within one week prior to consent.
  • Ongoing documented or suspected infection.
  • Severe cardiovascular disease that is a contraindication to terlipressin therapy such as a history of myocardial infarction, angina pectoris, advanced arteriosclerosis, uncontrolled cardiac arrhythmia, severe coronary insufficiency or uncontrolled hypertension.
  • Findings suggestive of severe organic renal disease (severe proteinuria/hematuria, or abnormal renal ultrasound suggestive of obstructive renal pathology).
  • Use of nephrotoxic drugs (e.g., aminoglycosides) in the 2 weeks before randomization
  • Severe comorbidity that in the opinion of the Investigator would affect short-term prognosis and/or disallow safe participation in the trial (such as for example, severe anemia or pancytopenia, advanced progressive neoplasia such as hepatocellular carcinoma, unless eligible for transplant).
  • Patients, who in the judgement of the Investigator, have been excessive alcohol drinkers in the past 12 weeks.
  • Patient is in the opinion of the investigator, despite previous education on sodium restriction, anticipated to remain grossly non-compliant of sodium restricted diet.
  • Recipient of renal or liver transplant.
  • Implanted alfapump or previous recipient of alfapump that had pump removed within past 3 months
  • Planned elective surgery related to cirrhosis complications, for example for hernia repair.
  • Known allergy or hypersensitivity to terlipressin.
  • Participation in other clinical research studies involving the treatment with other investigational drugs or evaluation with implantable devices within 30 days of consent.

    Eligibility last updated 4/14/22.  Questions regarding updates should be directed to the study team contact.

    Drug
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    Mayo Clinic — Rochester, MN

    Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

    Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

    David Dingli
    All
    Not specified
    This study is NOT accepting healthy volunteers
    0000-113845-P01-RST
    1582-02
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    .

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    Mayo Clinic — Rochester, MN

    A Prospective, Multi‐Center Study of the Medtronic Braive™ Growth Modulation System When Used in the Treatment of Pediatric Patients Diagnosed with Juvenile or Adolescent Idiopathic Scoliosis (BRAIVE IDE Study) (BRAIVE IDE)

    Study of the Braive Growth Modulation System for Progressive Pediatric Scoliosis (BRAIVE IDE)

    Annalise Larson
    All
    9 years to 16 years old
    This study is NOT accepting healthy volunteers
    2021-305386-P01-RST
    21-007823
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    Inclusion Criteria:

    • Has a diagnosis of juvenile or adolescent idiopathic scoliosis.
    • Is skeletally immature with a Sanders Score of ≥ 2 to ≤ 5.
    • Has failed conservative care as per investigator’s assessment.
    • Has a main thoracic Cobb angle between 30 and 60 degrees.
    • Has a Lenke Classification of 1A, 1B, or 1C.
    • Has kyphosis ≤ 40 degrees with a sagittal thoracic modifier N or negative.
    • Informed Consent Form/Assent and Authorization to Use and Disclose Health Information (if applicable) have been signed by parent/legal guardian and/or patient/participant per local requirement.


    Exclusion Criteria:

    • Has undergone previous spinal fusion procedure(s) at the affected levels.
    • Is pregnant or plans to become pregnant within the first 24‐months of the study.
    • Has a curve that requires instrumentation below L1.
    • Has spinal MRI abnormalities (e.g., CHIARI malformation, Syrinx greater than 4mm, tethered cord).
    • Has any type of non‐idiopathic scoliosis.
    • Has a left‐sided curve.
    • Has an associated syndrome.
    • Has a history of malignant hyperthermia.
    • Has an active or significant risk of infection (immunocompromised).
    • Has inadequate tissue coverage over the operative site as per investigator’s assessment.
    • Has a suspected or documented allergy or intolerance to implant materials.
    • Has a major psychiatric disorder/ history of drug abuse that would interfere with the subject’s ability to comply with study instructions or might confound the study interpretation as per investigator’s assessment (DSM‐5 can be used as a reference).
    • Is a ward of the court/state.
    • Has had prior ipsilateral or contralateral chest surgery.
    • Has severe chronic lung disease (e.g., asthma, bronchiectasis).
    • Has poor bone quality, as determined by the investigator, that may limit anterior fixation.
    • Is unwilling or unable to return for follow‐up visits and/or follow intra‐operative and/or postoperative instructions.
    • Concurrent participation in another clinical study that may add additional safety risks and/or confound study results*.
    • Subjects in concurrent studies can only be enrolled with permission from Medtronic. Please contact Medtronic’s study manager to determine if the subject can be enrolled in the BRAIVE IDE Study.

    *Subjects in concurrent studies can only be enrolled with permission from Medtronic. Please contact Medtronic’s study manager to determine if the subject can be enrolled in the BRAIVE IDE Study. 

    Eligibility last updated 11/16/21. Questions regarding updates should be directed to the study team contact.

     

     

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    Mayo Clinic — Rochester, MN

    Role of Biomarkers in Risk Stratification in Adults with Congenital Heart Disease: The ACHD Biobank Project

    A Study to Assess the Role of Biomarkers in Risk Stratification in Adults with Congenital Heart Disease

    Alexander Egbe
    All
    18 years and over
    This study is NOT accepting healthy volunteers
    0000-121771-H01-RST
    19-001161
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    Inclusion Criteria:

    • Adult patients (age ≥ 18 years)
    • Adult patients with diagnosis of Congenital Heart Disease (i.e., Tetralogy of Fallot (TOF), Pulmonary Atresia, Coarctation of Aorta, etc.).


    Exclusion Criteria:
     

    • Patients without research authorization.

    Eligibility last updated 11/10/21. Questions regarding updates should be directed to the study team contact.

     

     

     

     

    Coarctation of the aorta, Congenital heart defects in adults, Ebstein anomaly, Pulmonary atresia, Pulmonary valve stenosis, Tetralogy of Fallot, Transposition of the great arteries
    Atresia of pulmonary valve, Cardiovascular system, Coarctation of aorta, Congenital stenosis of pulmonary valve, Corrected transposition of great vessels, Ebstein's anomaly, History of creation of conduit of right atrium and pulmonary artery, L - transposition of the great vessels, Tetralogy of Fallot
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    Mayo Clinic — Rochester, MN

    Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

    This is a treatment study for a allogeneic hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD), thalassemia, Diamond Blackfan Anemia (DBA) and other non-malignant hematologic disorders that are transfusion dependent or represent other potentially life-threatening cytopenias. The objective of this study is to confirm the findings of our previous allogeneic hematopoietic stem cell transplant trial for non-malignant hematologic disorders that are transfusion dependent or represent other potentially life-threatening cytopenias including: • incidence of graft failure • disease free survival (DFS) including red cell transfusion independence at 6 months, 1 and 2 years • overall survival at 6 months, 1 and 2 years

    Ashish Gupta
    gupta461@umn.edu
    All
    up to 55 Years old
    N/A
    This study is NOT accepting healthy volunteers
    NCT02179359
    1407M52125
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    Inclusion Criteria:

    • Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
    • Acceptable stem cell source identified
    • Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
    • Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
    • Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
    • Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%
    Exclusion Criteria:

    • active, uncontrolled infection
    • pregnant or breastfeeding
    • HIV positive
    Drug: Reduced Toxicity Ablative Regimen, Drug: Reduced Intensity Preparative Regimen, Drug: Myeloablative Preparative Regimen
    Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
    Stem Cell Transplant
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    University of Minnesota Medical Center, Fairview — Minneapolis, Minnesota Lisa Burke - (lburke3@Fairview.org)

    Prophylaxis Regimen for Hemophilia A Patients (PREDICT)

    All
    12 Years and over
    Phase 4
    This study is NOT accepting healthy volunteers
    NCT05036278
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    Inclusion Criteria:

    • Participants must be ≥ 12 years of age inclusive, at the time of signing the informed consent/assent.
    • Previously treated patients (≥ 150 EDs) with congenital hemophilia A.
    • Prophylaxis with any SHL FVIII product with a stable frequency for at least 6 consecutive months within the last 12 months prior to screening before entering the study and documented in medical records. Stable frequency is defined as a minimum 18 weeks of treatment in a 6 (consecutive) calendar month period in the 12 months prior to screening. Patients can be on any non-Jivi EHL between the 6-month stable SHL prophylaxis period and start of study treatment.
    • Documented bleeding rate (ABR) while on stable frequency SHL prophylaxis for at least 6 consecutive months within the last 12 months prior to screening.
    • No current evidence (≥ 0.6 BU/mL) of FVIII inhibitors. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL on two occasions) but has been tolerized for at least 1 year since the last positive titer with at least 1 negative inhibitor assay test during that period, they can be enrolled. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL) but did not require tolerization and has had at least 1 negative inhibitor assay test during a minimum period of at least 1 year since the last positive titer, they can be enrolled.
    • If they are human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4+) lymphocyte count should be > 200/mm^3 within 1 year before entering the study and documented in medical records. -
    • Participants who are willing to complete an electronic diary (eDiary).
    • Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
    • For adolescent participants (≥ 12 to < 18 years), a legal guardian must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Activities (SoA) (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the PROs during the scheduled study visits; accurately and reliably dispense study intervention as directed.
    • For adolescent participants, a legal guardian must be able to accurately maintain the child's take-home record, including items of general health.
    Exclusion Criteria:

    • Any other inherited or acquired bleeding disorder in addition to hemophilia A. Note: von Willebrand disease should be diagnosed per local clinical practice. Participants with a diagnosis of von Willebrand disease in medical records or diagnosed at the time of screening will be excluded.
    • Platelet count < 100,000/mm^3
    • Evidence of inhibitor to FVIII (≥ 0.6 BU/mL) within the last 1 year
    • The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug or device within 30 days of signing informed consent.
    • The participant has a planned major surgery.
    • Documentation of missing risk score parameters other than physical activity .
    • Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.
    • Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study.
    • Close affiliation with the investigational site; e.g. a close relative of the investigator, dependent person (e.g. employee or student of the investigational site).
    • Otherwise vulnerable participants (e.g. participants who are in custody by order of an authority).
    • Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures (i.e. eDiary completion, clinic visits, phone updates), restrictions, and requirements.
    Biological: Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
    Hemophilia A, Prophylaxis of Bleeding
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    University of Minnesota Medical Center — Minneapolis, Minnesota

    MOR208C310: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Trial Comparing the Efficacy and Safety of Tafasitamab Plus Lenalidomide in Addition to R-CHOP Versus R-CHOP in Previously Untreated, High-intermediate and High-risk Patients With Newly-diagnosed Diffuse Large B-cell Lymphoma (DLBCL) (frontMIND)

    Tafasitamab + Lenalidomide + R-CHOP Versus R-CHOP in Newly Diagnosed High-intermediate and High Risk DLBCL Patients

    Grzegorz Nowakowski
    All
    18 years to 80 years old
    Phase 3
    This study is NOT accepting healthy volunteers
    2021-303502-P01-RST
    21-001047
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    Inclusion Criteria:

    • Written informed consent.
    • Age 18 to 80 years at time of signing of the ICF.
    • Previously untreated patients with local biopsy-proven, CD20-positive DLBCL, including one of the following diagnoses by 2016 World Health Organization (WHO) classification of lymphoid neoplasms are eligible: 
      • DLBCL, NOS including GCB type, ABC type;
      • T-cell rich large BCL;
      • Epstein-Barr virus-positive DLBCL, NOS;
      • Anaplastic lymphoma kinase (ALK)-positive large BCL;
      • Human herpes virus-8 (HHV8)-positive DLBCL, NOS;
      • High-grade BCL with MYC and B-cell lymphoma 2 (BCL2) and/or B-cell lymphoma 6 (BCL6) rearrangements (double-hit or triple-hit lymphoma);
      • Note: Patients must be appropriate candidates for R-CHOP. If an investigator deems a patient with a known double- or triple-hit lymphoma (HGBL) should be treated more aggressively (e.g., dose-adjusted etoposide, prednisone, vincristine, cyclophosphamide, doxorubicin and rituximab [DA-EPOCH-R] or cyclophosphamide, vincristine, doxorubicin and dexamethasone (CVAD) followed by methotrexate and cytarabine [Hyper CVAD]), this patient would not be considered eligible for this study;
      • DLBCL coexistent with either follicular lymphoma (FL) of any grade, gastric MALT lymphoma or non-gastric MALT lymphoma;
      • FL grade 3b.
    • Availability of archival or freshly collected tumor tissue sent for retrospective central pathology review.
    • IPI status of 3 to 5 (for patients > 60 years of age) or aaIPI 2 to 3 (for patients ≤ 60 years of age).
    • Diagnosis to treatment interval, defined as the time between the date of DLBCL diagnosis (date of the first biopsy specimen containing lymphoma according to the local pathology report) and the start of treatment (C1D1) ≤ 28 days.
    • ECOG performance status of 0, 1, or 2.
    • Left ventricular ejection fraction equal to or greater than lower limit of institutional normal range, assessed by local echocardiography or cardiac multi-gated acquisition (MUGA) scan.
    • Adequate hematologic function.
    • Female participants: Agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods and refrain from breast feeding and donating eggs; agreement to ongoing pregnancy testing during the course of the study, and after study therapy has ended.
    • Male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom and agreement to refrain from donating sperm.


    Exclusion Criteria:

    • Any other histological type of lymphoma according to WHO 2016 classification of lymphoid neoplasms; e.g., primary mediastinal (thymic) large B-cell lymphoma, Burkitt's lymphoma, BCL, unclassifiable, with features intermediate between DLBCL and classical Hodgkin lymphoma (grey-zone lymphoma); primary effusion lymphoma; primary cutaneous DLBCL, leg type; primary DLBCL of the CNS; DLBCL arising from CLL or indolent lymphoma.
    • History of prior non-hematologic malignancy except for the following:
      • Malignancy treated with curative intent and with no evidence of active disease present for more than 2 years before screening;
      • Adequately treated lentigo maligna melanoma without current evidence of disease or adequately controlled non-melanomatous skin cancer;
      • Adequately treated carcinoma in situ without current evidence of disease;
      • Any systemic anti-lymphoma and/or investigational therapy prior to the start of C1D1, except for permitted pre-phase treatment;
      • Contraindication to any of the individual components of R-CHOP, including prior receipt of anthracyclines;
      • Known CNS lymphoma involvement;
      • Known active systemic bacterial, viral, fungal, or other infection at screening, including patients with suspected active or latent tuberculosis (as confirmed by a positive interferon-gamma release assay);
      • Pregnancy or lactation;
      • History or evidence of clinically significant cardiovascular, CNS and/or other systemic disease that in the investigator's opinion would preclude participation in the study or compromise the patient's ability to give informed consent.
    Drug, Other, Behavioral
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    Mayo Clinic — Rochester, MN

    TrAstuzumab Cardiomyopathy Therapeutic Intervention with Carvedilol (TACTIC) Trial (TACTIC)

    Treating Breast Cancer Patients Undergoing Trastuzumab Treatment with Carvedilol to Reduce Incidence of Heart Failure

    Joerg Herrmann
    Female
    18 years and over
    Phase 2, Post Market
    This study is NOT accepting healthy volunteers
    0000-101043-P01-RST
    18-006090
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    Inclusion Criteria:

    • ≥ 18 years of age.
    • New or locally recurrent diagnosis of HER2+ breast cancer that will be treated with curative intent.
    • Planned HER2-directed therapy (any therapy targeting HER2 signaling including trastuzumab +/- pertuzumab or trastuzumab-emtansine (T-DM1)). Neratinib and lapatinib will not be considered “Her2-directed therapy” or “anti-HER2” for the purpose of this study.


    Exclusion Criteria:

    • Metastatic breast cancer (distant metastases).
    • Active systemic treatment for non-breast cancer.
    • History of HF of any class and type, or diagnosis of cardiomyopathy in the past, LVEF < 50% at screening, intolerance to beta-blocker, baseline use of any beta-blocker for coronary artery disease including myocardial infarction, current ACE inhibitor or ARB therapy for hypertension in the presence of diabetes and/or for chronic kidney disease/proteinuria, on active therapy with amiodarone, sotalol, or any other antiarrhythmic history of bronchial asthma or related  bronchospastic conditions, heart rate < 50 BPM at screening (average of 3 most recent readings).
    • History of or current sick sinus syndrome.
    • AV block grade II or higher (unless patient has a permanent pacemaker) at screening.
    • Systolic blood pressure < 90 mmHg at screening (average of 3 most recent readings).
    • Severe hepatic dysfunction, as defined by NCI ODWG (total bilirubin > 3x ULN with any AST elevation) or Child Pugh C class.
    • Pregnancy.
    Device, Drug, Administration of antineoplastic agent, Drug therapy, Primary prevention of cardiovascular disease
    Breast cancer, Cancer, Heart failure
    Cancer treatment, Cardiovascular disease prevention, Cardiovascular system, Chemotherapy, Chemotherapy for breast cancer, HER2-positive carcinoma of breast, Heart failure as a complication of care, Medical Oncology, carvedilol, trastuzumab
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    Mayo Clinic — Rochester, MN

    Detection of Esophageal Cancer by Assay of Novel Methylated DNA Markers in Plasma, a Phase II Study

    Detection of Esophageal Cancer by Assay of Novel Methylated DNA Markers in Plasma

    John Kisiel
    All
    18 years and over
    This study is NOT accepting healthy volunteers
    0000-120879-H01-RST
    18-004438
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    Study Criteria:

    Exclusions:

    • Age < 18
    • Primary cancer outside of the esophagus within the last 5 years (not including basal or squamous cell skin cares)
    • History of Esophageal cancer
    • Intervention to completely remove the current target pathology
    • Any endoscopic treatment to current target pathology [Endoscopic intervention examples: Photodynamic therapy, Halo, Cryotherapy, Multi-polar coagulation]
    • Prior chemotherapy class drugs or radiation for the primary esophageal cancer prior to blood or stool collection
    • Biopsy (excluding skin) and/or brushing 3 days prior to blood collection  
    • IV contrast given within 24 hours of blood collection

     

    Additional Stool Exclusions:

    • Bowel prep <7 days prior to stool collection
    • Oral contrast given within 7 days of stool collection
    • Removal of more than 50% of colon or presence of ileostomy
    • Enteral feeds or TPN
    • Diagnosis of inflammatory bowel disease
    • If patient has had colonoscopy within 5 years, were pre-cancerous polyps (>1cm or villous)
    • Enteric barium studies within the prior 7 days of stool collection
    Cancer, Esophageal cancer, Squamous cell carcinoma of the skin
    Adenocarcinoma of esophagus, Digestive system, Integumentary system, Medical Oncology, Squamous cell carcinoma of esophagus
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    Mayo Clinic — Rochester, MN

    Identification of Prognostic Biomarkers for Progression of Invasive Squamous Cell Carcinoma

    A Study Identifying Predictive Biomarkers for the Progression of Invasive Squamous Cell Carcinoma

    Dennis Wigle
    All
    18 years and over
    This study is NOT accepting healthy volunteers
    0000-117257-H01-RST
    15-000548
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    Inclusion Criteria

    • known or suspected squamous cell carcinoma of the lung
    • able to provide consent
    • could have already had a bronchoscopy or surgical resection with tissue saved at the Mayo Clinic Rochester
    • will be undergoing a bronchoscopy and having a surgical resection at the Mayo Clinic Rochester

     

    Exclusion Criteria

    • unable to provide consent
    • pregnant

     

    Cancer, Lung cancer, Squamous cell carcinoma of the skin
    Bronchoscopy, Integumentary system, Medical Oncology, Respiratory system, Squamous cell carcinoma of bronchus, Squamous cell carcinoma of lung
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    Mayo Clinic — Rochester, MN

    Acute Detection of Non-convulsive Seizures with Single-channel EEG

    A Study to Detect Non-convulsive Seizures with Single-channel Electroencephalogram (EEG)

    Alejandro Rabinstein
    All
    18 years and over
    This study is NOT accepting healthy volunteers
    0000-122067-H01-RST
    19-003718
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    Inclusion Criteria:

    • Patients admitted with encephalopathy concerning for non-convulsive seizures or NCSE who can also undergo conventional multi-lead EEG monitoring.
    • ≥ 18 years of age.


    Exclusion Criteria:
     

    • Patients who have dermatologic contraindication to the adhesive by which the portable EEG device is attached.
    • < 18 years of age.

     

    Encephalopathy, Seizure
    Disorder of brain, EEG, Generalized seizure, Nervous system, Nonconvulsive status epilepticus
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    Mayo Clinic — Rochester, MN

    Randomized Phase II Stereotactic Ablative Radiation Therapy (SABR) for Metastatic Unresected Renal Cell Carcinoma (RCC) Receiving Immunotherapy (SAMURAI) (SAMURAI)

    Testing the Addition of Stereotactic Radiation Therapy With Immune Therapy for the Treatment of Patients With Unresectable or Metastatic Renal Cell Cancer, SAMURAI Study

    Mark Waddle
    All
    18 years and over
    Phase 2
    This study is NOT accepting healthy volunteers
    2022-309256-P01-RST
    22-012786
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    Inclusion Criteria:


    - Pathologically (histologically or cytologically) proven diagnosis of renal cell
    carcinoma prior to registration

    - Node-positive unresectable (TxN1Mx) or metastatic (TxNxM1) based on the following
    diagnostic workup:

    - History/physical examination within 45 days prior to registration

    - CT/magnetic resonance imaging (MRI) of the chest/abdomen/pelvis within 45 days
    prior to registration

    - Patients must have IMDC intermediate (1-2 factors) or poor risk disease (>= 3 factors)

    - Patients with a prior or concurrent malignancy whose natural history or treatment does
    not have the potential to interfere with the safety or efficacy assessment of the
    investigational regimen are eligible for this trial

    - Patients with measurable disease (node positive or metastatic) as defined by RECIST
    version 1.1 excluding the primary renal tumor

    - Patient not recommended for or refused immediate cytoreductive nephrectomy

    - Candidate for standard of care therapy with either immuno-oncology (IO)-IO or IO-VEGF
    combination regimen

    - Primary renal tumor measuring 8 cm or less in anterior to posterior dimension only on
    axial imaging

    - Age >= 18

    - Karnofsky performance status >= 60 within 45 days prior to registration

    - Hemoglobin >= 8 g/dL (transfusions are allowed) (within 45 days prior to registration)

    - Platelet count >= 50,000/mm^3 (within 45 days prior to registration)

    - Absolute neutrophil count (ANC) >= 1500/mm^3 (within 45 days prior to registration)

    - Calculated (Calc.) creatinine clearance >= 30 mL/min (within 45 days prior to
    registration)

    - For African American patients specifically whose renal function is not considered
    adequate by the formula above, an alternative formula that takes race into
    account (Chronic Kidney Disease Epidemiology Collaboration CKD-EPI formula)
    should be used for calculating the related estimated glomerular filtration rate
    (GFR) with a correction factor for African American race creatinine clearance for
    trial eligibility, where GFR >= 30 mL/min/1.73m^2 will be considered adequate

    - Total bilirubin =< 1.5 x upper limit of normal (ULN) (except subjects with Gilbert
    Syndrome, who can have total bilirubin < 3.0 mg/dL) (within 45 days prior to
    registration)

    - Aspartate aminotransferase and alanine aminotransferase (AST and ALT) =< 3 x upper
    limit of normal (ULN) or < 5 x ULN if hepatic metastases present (within 45 days prior
    to registration)

    - Patients with known human immunodeficiency virus (HIV) on effective anti-retroviral
    therapy with undetectable viral load within 6 months are eligible for this trial.
    Testing is not required for entry into protocol

    - For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral
    load must be undetectable on suppressive therapy, if indicated

    - Patients with a history of hepatitis C virus (HCV) infection must have been treated
    and cured. Patients with HCV infection who are currently on treatment are eligible if
    they have an undetectable HCV viral load

    - The patient must agree to use a highly effective contraception, including men with
    vasectomies if they are having sex with a woman of childbearing potential or with a
    woman who is pregnant, while on study drug and for 6 months following the last dose of
    study drug. Childbearing potential is defined as any person who has experienced
    menarche and who has not undergone surgical sterilization (hysterectomy or bilateral
    oophorectomy) or who is not postmenopausal

    - The patient or a legally authorized representative must provide study-specific
    informed consent prior to study entry and, for patients treated in the United States
    (U.S.), authorization permitting release of personal health information


    Exclusion Criteria:


    - Patients with planned treatment of all metastatic disease with definitive therapy
    including either surgery, ablative (non-palliative) doses of radiation, or
    intervention of some type (definitive interventional radiology techniques) to ALL
    metastatic sites rendering the patient without extra-renal measurable disease.
    Patients NOT planned for definitive treatment of all metastatic sites are eligible.
    Lesions radiated palliatively are not eligible for response assessment

    - Patients with untreated or unstable brain metastases or cranial epidural disease

    - Note: Patients who have been adequately treated with radiotherapy, radiosurgery,
    or surgery and stable for at least 4 weeks prior to registration as documented by
    MRI or CT imaging or deemed stable by clinical investigator are eligible. Treated
    brain metastases are defined as having no ongoing requirement for steroids and no
    evidence of progression or hemorrhage after treatment for at least 4 weeks prior
    to registration as documented by MRI or CT imaging or deemed stable by clinical
    investigator

    - Prior radiotherapy to the kidney that would result in overlap of radiation therapy
    fields treatment of the primary tumor

    - Any prior systemic therapy for metastatic renal cell carcinoma (RCC) note that prior
    chemotherapy for a different cancer is allowed (completed > 3 years prior to
    registration)

    - Severe, active comorbidity defined as follows:

    - Active autoimmune disease requiring ongoing therapy including systemic treatment
    with corticosteroids (> 10 mg daily prednisone equivalents) or other
    immunosuppressive medications daily. Inhaled steroids and adrenal replacement
    steroid doses > 10 mg daily prednisone equivalents are permitted in the absence
    of active autoimmune disease

    - History of severe allergic, anaphylactic or other hypersensitivity reactions to
    chimeric or humanized antibodies

    - Active tuberculosis (purified protein derivative [PPD] response without active
    tuberculosis [TB] is allowed)

    - Uncontrolled hypertension (systolic blood pressure [BP] > 190 mmHg or diastolic
    BP > 110 mmHg)

    - Major surgery < 45 days prior to registration.

    - Any serious (requiring hospital stay or long term rehab) non-healing wound,
    ulcer, or bone fracture within 45 days prior to registration

    - Any arterial thrombotic (ST elevation myocardial infarction [STEMI], non-ST
    elevation myocardial infarction [NSTEMI], cerebrovascular accident [CVA], etc)
    events within 180 days prior to registration

    - Active New York (NY) Heart Association class 3-4 heart failure symptoms

    - Moderate or severe hepatic impairment (Child-Pugh B or C)

    - Any history of untreated pulmonary embolism or deep venous thrombosis (DVT)
    within 180 days prior to registration. (Any asymptomatic or treated pulmonary
    embolism or asymptomatic treated deep venous thrombosis > 30 days prior to
    registration is allowed)

    - Unstable cardiac arrhythmia within 180 days prior to registration

    - History of abdominal fistula, gastrointestinal perforation, intra-abdominal
    abscess, bowel obstruction, or gastric outlet obstruction within 180 days prior
    to registration

    - History of or active inflammatory bowel disease

    - Malabsorption syndrome within 45 days prior to registration

    - Pregnancy and individuals unwilling to discontinue nursing. For women of child bearing
    potential must have a negative pregnancy test =< 45 days prior to registration

    Biologic/Vaccine, Drug
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    Mayo Clinic — Rochester, MN

    A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over

    A Dose-Ranging Study to Evaluate the Effectiveness and Safety of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

    Megha Tollefson
    All
    6 years to 65 years old
    Phase 2/3
    This study is NOT accepting healthy volunteers
    0000-121772-P01-RST
    19-001189
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    Inclusion Criteria:

    • Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained.
    • Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma.
    • An FA severity score of 2 or 3 on the IGA scale.
    • Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation.
    • Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator.


    Exclusion Criteria:

    • Patients who cannot carry out the treatment plan or follow-up assessment.
    • Patients with serious skin lesions such as erosions or ulcers.
    • Patients with known hypersensitivity to any component of the study product.
    • Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment.
    • Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment.
    • Patients who participated in any other clinical trial within 3 months prior to the day of enrolment.
    • Patients judged unsuitable for this clinical trial by the investigator or sub-investigator.
    • Pregnant or lactating females.
    • Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception.
    • Patients with immune dysfunction or receiving any form of immunosuppression.
    • Patients with severe FA, with a score of 4 on the IGA scale.
    • Patients with an FA severity score of less than 2 on the IGA scale.
    Drug, Administration of drug or medicament to skin via topical route, Drug therapy
    Tuberous sclerosis
    Fibrous skin tumor of tuberous sclerosis, Tuberous sclerosis syndrome, sirolimus
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    Mayo Clinic — Rochester, MN

    A Phase 3 Study of the Efficacy and Safety of Lixivaptan in Participants with Autosomal Dominant Polycystic Kidney Disease Consisting of a 1-year Double-blind, Placebo-controlled, Randomized Phase and a 1-year Open-Label Phase: The ACTION Study (ACTION 301)

    Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease

    Vicente Torres
    All
    18 years to 60 years old
    Phase 3
    This study is NOT accepting healthy volunteers
    2021-306070-P01-RST
    21-010236
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    Inclusion Criteria:


    - Diagnosis of ADPKD by appropriate imaging or genetic testing

    - Mayo Clinic MRI imaging classification of 1C, 1D or 1E

    - eGFR ≥25 mL/min/1.73 m2 and ≤90 mL/min/1.73 m2

    - Body mass index (BMI) between 18 and 40 kg/m2

    - Control of hypertension consistent with KDIGO guidelines without a diuretic

    - Willing to practice acceptable methods of birth control (both males who have partners
    of child-bearing potential and females of childbearing potential)


    Exclusion Criteria:


    - Known sensitivity or idiosyncratic reaction to any compound present in lixivaptan and
    related compounds.

    - Hypovolemia or inability to perceive thirst

    - Abnormal serum sodium concentration at Screening

    - Subjects who have taken any investigational drug or used an investigational device
    within 30 days, or 5 half-lives, whichever is longer, prior to Screening

    - Subjects who are taking, have taken within the past 2 weeks, or are expected to be
    taking, strong or moderate CYP3A4 or CYP2C8 inhibitors or inducers including regular
    use of grapefruit juice or Seville oranges

    - Prior use of tolvaptan or lixivaptan within the past 2 months.

    - Prior use of conivaptan, somatostatin analogs (e.g. lanreotide, pasireotide,
    octreotide, etc.), metformin, nicotinamide, bardoxolone, venglustat, demeclocycline,
    or mammalian target of rapamycin (mTOR) kinase inhibitors (e.g. everolimus, sirolimus,
    etc.) to treat ADPKD within the past 2 months

    - Prior use of a sodium-glucose cotransporter 2 (SGLT2) inhibitor (e.g., canagliflozin,
    dapagliflozin, empagliflozin, etc.) within the past 2 months or expected need for
    initiation of treatment with a SGLT2 inhibitor during the study.

    - Prior use of a hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor within
    the past 2 months or expected need for initiation of treatment with a HIF-PH inhibitor
    during the study.

    - Requirement for chronic diuretic use

    - Advanced diabetes (e.g., glycosylated hemoglobin [HgbA1c] >7.5%, and/or glycosuria by
    dipstick, significant proteinuria [>300 mcg albumin/mg creatinine]), other significant
    renal disease, renal cancer, transplanted kidney, single kidney, recent kidney surgery
    within the past 6 months (including cyst drainage or fenestration) or acute kidney
    injury within past 6 months

    - Clinically significant incontinence, overactive bladder, or urinary retention (e.g.,
    benign prostatic hyperplasia).

    - New York Heart Association Functional Class 3 or 4 heart failure or other significant
    cardiac or electrocardiogram (ECG) findings that could pose a safety risk to the
    subject.

    - Positive test results for hepatitis B surface antigen (HBsAg) or hepatitis C (HCV).

    - History of infection with human immunodeficiency virus (HIV) unless the participant is
    stable and doing well on a non-CYP interacting anti-retroviral therapy (ART) regimen
    and who has not required more than 2 changes in their ART regimen since treatment
    inception.

    - History of clinically significant drug or alcohol abuse in the past 2 years.

    - Contraindication to or interference with MRI assessments.

    - Malignancy within the past 5 years except for those not considered to affect
    participant survival.

    Note: Other protocol defined Inclusion/Exclusion criteria may apply.

    Eligibility last updated 6/15/22.  Questions regarding updates should be directed to the study team contact.

    Drug
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    Mayo Clinic — Rochester, MN

    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Evaluate the Safety and Efficacy of Eluxadoline in Pediatric Patients (Age 12 to 17 Years) With Irritable Bowel Syndrome With Diarrhea (IBS-D)

    Study to Explore the Therapeutic Effect of Eluxadoline in Treating Irritable Bowel Syndrome With Diarrhea in Children

    Mhd Louai Manini
    All
    12 years to 17 years old
    Phase 2
    This study is NOT accepting healthy volunteers
    0000-120204-P01-RST
    17-008759
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    Inclusion Criteria:

    • Patient must provide written or verbal informed assent and the parent/guardian/LAR must provide written informed consent before the initiation of any study-specific procedures.
    • Patient is a male or female outpatient, 12 to 17 years of age inclusive, at the time the patient provides assent for the study and parent/guardian/LAR has provided signed consent.
    • Patient is able to read and understand the assessments in the eDiary.
    • Female patients of childbearing potential must have a negative serum pregnancy test at Visit 1 (screening) and a negative urine pregnancy test at Visit 3 (randomization) prior to dosing.
    • Female patients who have had their first menstrual period and are sexually active must agree to use a reliable form of contraception. Reliable contraception is defined as:
      • Hormonal contraception (eg, oral contraceptive, contraceptive implant, or injectable hormonal contraceptive).
      • Double-barrier method (eg, condom plus intrauterine device, diaphragm plus spermicide).
    • Patient has a diagnosis of IBS-D as defined by the modified Rome IV child/adolescent criteria*: Must include all of the following:
      •  Abdominal pain at least 4 days per month over at least 2 months associated with one or more of the following:
        •  Related to defecation
        •  A change in frequency of stool
        •  A change in form (appearance) of stool
      • After appropriate evaluation, the symptoms cannot be fully explained by another medical condition
      • Patient has predominantly diarrheal stool symptoms defined as Bristol stool types 6 or 7 for more than 25% of bowel movements and Bristol stool types 1 or 2 for less than 25% of bowel movements that occur in the absence of laxatives
    • All criteria fulfilled for at least 2 months prior to Visit 1 (screening). 
    • Patient has been compliant with the eDiary by completing both the morning and evening assessments for at least 8 out of the 14 days immediately preceding Visit 3 (randomization).
    • Patient has an average daytime abdominal pain scoreless than or equal to 2.0 over the 2 weeks prior to randomization. 
    • Patient has at least 1 daytime bowel movement with a consistency of Type 6 or Type 7 on the pediatric Bristol Stool Form Scale (p-BSFS) on at least 2 days per week during the 2 weeks prior to randomization that occurs in the absence of laxatives. 
    • Patient has no clinically significant findings on a physical examination, vital sign assessment, electrocardiogram (ECG), and clinical laboratory tests (clinical chemistry panel, complete blood count, urine drug screen, urinalysis) after providing informed assent and after written consent is obtained, but before receiving the first dose of study treatment. (A central laboratory will be used to evaluate all urine [except urine pregnancy tests] and blood samples and will utilize reference ranges specific to a patient's age and gender. ECGs will be performed and electronically transmitted to a central ECG laboratory for analysis by a pediatric cardiologist in accordance with the instructions provided by the central ECG laboratory. The Investigator will determine if a particular finding is clinically significant. [In making this determination, the Investigator will consider whether the particular finding could represent a condition that would exclude the patient from the study, could represent a safety concern if the patient participates in the study, or could confound the study-specific assessments of safety or efficacy.])


    Exclusion Criteria:

    • Patient has no gallbladder, (ie, agenesis of the gallbladder or cholecystectomy). 
    • Patient has had any of the following surgeries:
      • Any abdominal surgery within the 3 months prior to Screening; or
      • A history of major gastric, hepatic, pancreatic, or intestinal surgery.  (Note: appendectomy, hemorrhoidectomy, or polypectomy greater than 3 months post-surgery are allowed. For the purposes of this study, laparoscopic surgeries without complication are considered minor and non-exclusionary, provided the condition for which the surgery was performed was not exclusionary.)
    • Patient has a history of chronic or severe constipation or sequelae from constipation, or known or suspected mechanical GI obstruction or pseudo obstruction. 
    • Patient has a history or current diagnosis of constipation with encopresis. 
    • Patient meets the child/adolescent Rome IV criteria of IBS with constipation, IBS with constipation and diarrhea (mixed), unspecified IBS, or Patient has a history of intestinal obstruction, stricture, toxic megacolon, GI perforation, fecal impaction, gastric banding, bariatric surgery, adhesions, ischemic colitis, or impaired intestinal circulation. 
    • Patient has a documented history of hepatic impairment as defined by Child-Pugh Classification Grade A, B or C. 
    • Patient has a history or current diagnosis of inflammatory or immune-mediated GI disorders including inflammatory bowel disease (ie, Crohn's disease, ulcerative colitis, microscopic colitis). 
    • Patient has celiac disease, or a positive serological test for celiac disease and the condition has not been ruled out by endoscopic biopsy.
    • Patient has any congenital and/or acquired malabsorption syndrome (eg, Shwachman-Diamond syndrome). 
    • Patient has a history of a microbiologically documented (ie, stool culture or medical history) GI infection within 3 months prior to Screening. 
    • Patient has a known lactose or fructose intolerance that is associated with diarrhea, abdominal pain or discomfort, and that could confound assessments in the study. 
    • Patient has a history of diverticulitis within 3 months prior to Screening.
    Drug, Drug therapy
    Irritable bowel syndrome
    Digestive system, Irritable bowel syndrome variant of childhood with diarrhea, Irritable bowel syndrome with diarrhea, eluxadoline
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    Mayo Clinic — Rochester, MN

    A Randomized Phase 2/3 Study of Olaparib Plus Temozolomide Versus Investigator's Choice for the Treatment of Patients With Advanced Uterine Leiomyosarcoma After Progression on Prior Chemotherapy

    Testing Olaparib and Temozolomide Versus the Usual Treatment for Uterine Leiomyosarcoma After Chemotherapy Has Stopped Working

    Brittany Siontis
    All
    18 years and over
    Phase 2/3
    This study is NOT accepting healthy volunteers
    2021-304188-P01-RST
    23-002999
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    Inclusion Criteria:


    - Histologically confirmed leiomyosarcoma of uterine origin, as established by the site
    enrolling the patient on study. Central pathology review will not occur.

    - Metastatic or locally advanced and surgically unresectable disease, in the opinion of
    the treating investigator.

    - Patients must have at least one lesion that is measurable per Response Evaluation
    Criteria in Solid Tumors (RECIST) version (v)1.1 to be eligible for the study.

    - Women of childbearing potential only, a negative pregnancy test done ≤ 7 days prior to
    registration is required.

    - Age ≤ 18 years.

    - Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2.

    - Patients must have had prior progression on, or intolerance to, at least two prior
    lines of systemic therapy for advanced uLMS, one of which was an anthracycline
    (anthracycline monotherapy or combination). Adjuvant chemotherapy will qualify as a
    prior line of treatment. Endocrine treatment will not qualify as a prior line of
    treatment.

    - Patients must have recovered to baseline or ≤ grade 1 per CTCAE version 5.0 from
    toxicity related to any prior treatment, unless adverse events are clinically
    nonsignificant and/or stable on supportive therapy, with the exception of fatigue
    (which must be ≤ grade 2), alopecia and/or endocrinopathies related to prior
    immunotherapy which are controlled with hormone replacement.

    - Patients must have completed all prior anti-cancer treatment, including radiation, ≤
    28 days prior to registration.

    - Absolute neutrophil count (ANC) ≥ 1500/mm^3 (within ≤ 28 days prior to registration)

    - Platelet count ≥ 100,000/mm^3 (within ≤ 28 days prior to registration).

    - Creatinine ≤ 1.5 * upper limit of normal (ULN) (within ≤ 28 days prior to
    registration).

    * If creatinine > 1.5 * ULN, then creatinine clearance (CrCl) must be > 50 mL/min, per
    Cockcroft-Gault method.

    - Hemoglobin ≥ 9 g/dL (within ≤ 28 days prior to registration).

    * No transfusions ≤ 14 days before cycle 1 day 1 (C1D1).

    - Total bilirubin ≤ 1.5 x ULN (within ≤ 28 days prior to registration).

    * If documented Gilbert's: ≤ 2.0 x ULN.

    - Aspartate aminotransferase/alanine aminotransferase (AST/ALT) ≤ 3 x ULN (within ≤ 28
    days prior to registration).

    - For patients with evidence of chronic hepatitis B (HBV) infection, the HBV viral load
    must be undetectable on suppressive therapy, if indicated.

    - Patients with a history of hepatitis C virus (HCV) infection must have been treated
    and cured. For patients with HCV infection who are currently on treatment, they are
    eligible if they have an undetectable HCV viral load.

    - HIV-infected patients on effective anti-retroviral therapy with undetectable viral
    load within 6 months are eligible for this trial.

    - Patients with a prior or concurrent malignancy whose natural history or treatment does
    not have the potential to interfere with the safety or efficacy assessment of the
    investigational regimen are eligible for this trial.

    - Patients with central nervous system (CNS)/leptomeningeal disease must have undergone
    definitive treatment, have no evidence of CNS progression on follow-up imaging
    performed at least 4 weeks after the CNS-directed therapy is completed, and be off all
    steroids, in order to be eligible.

    - Patients must be able to swallow oral medications.

    - In order to complete the mandatory patient-completed measure, participants must be
    able to speak and/or read English and Spanish.

    - For all patients, prior to randomization and as part of eligibility, the investigator
    must select the agent which the patient would receive if assigned to the
    investigator's choice arm, prior to randomization. The patient must meet all
    eligibility criteria for that agent during screening and prior to randomization.

    - Patients without central venous access must be willing to undergo placement of central
    venous access (i.e., port or peripherally inserted central catheter (PICC) line, per
    institutional practice). if assigned to the investigator's choice arm and if the
    investigator intends to treat the patient with trabectedin. The site must be able to
    place central venous access within 10 days of registration/randomization.

    - In order to complete the mandatory patient-completed measure, participants must be
    able to speak and/or read English and Spanish

    - For all patients, prior to randomization and as part of eligibility, the investigator
    must select the agent which the patient would receive if assigned to the
    investigator's choice arm, prior to randomization. The patient must meet all
    eligibility criteria for that agent during screening and prior to randomization.
    Patients without central venous access must be willing to undergo placement of central
    venous access (i.e. port or peripherally inserted central catheter [PICC] line, per
    institutional practice). if assigned to the investigator's choice arm and if the
    investigator intends to treat the patient with trabectedin. The site must be able to
    place central venous access within 10 days of registration/randomization


    Exclusion Criteria:

    - Not pregnant and not nursing, because this study involves agents that
    have known genotoxic, mutagenic and teratogenic effects. Therefore, for women of
    childbearing potential only, a negative pregnancy test done ≤ 7 days prior to registration
    is required.

    - Patients may not have received prior treatment with any PARP inhibitor, temozolomide
    or dacarbazine (IV analogue of temozolomide).

    - Patients may not have had prior treatment with at least one of the agents included on
    the investigator's choice arm: trabectedin or pazopanib. If the patient has had prior
    treatment with one of these agents, they must be assigned to the other agent for
    investigator's choice. That is, patients who have received prior pazopanib must be
    assigned to trabectedin, and patients who have received prior trabectedin must be
    assigned to pazopanib.

    - Patients may not have undergone major surgery (related or unrelated to their cancer
    diagnosis) ≤ 28 days of registration. Subjects with clinically relevant ongoing
    complications from prior surgery are not eligible.

    - Patients may not have uncontrolled hypertension defined as a blood pressure (BP) >
    150/90 on two consecutive assessments during the screening period. If a patient is
    found to have a BP > 150/90 on two consecutive assessments during the screening
    period, the patient may be started on an anti-hypertensive regimen, and will be
    considered eligible if two subsequent measurements are performed and the BP is ≤
    150/90.

    - Patients may not have an uncontrolled ventricular arrhythmia or recent (within 3
    months) myocardial infarction.

    - In addition to the above, patients with known history or current symptoms of cardiac
    disease, or history of treatment with cardiotoxic agents, should have a clinical risk
    assessment of cardiac function using the New York Heart Association Functional
    Classification. To be eligible, patients should be class 2B or better.

    - Patients may not have a history of active or unresolved: perforation, abscess or
    fistula within 28 days prior to registration (either clinically or radiographically).

    - Patients must not have myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) or
    a history of bone marrow biopsy findings at any time consistent with MDS and/or AML.

    - Patients must not have an uncontrolled intercurrent illness including, but not limited
    to, ongoing or active infection, uncontrolled major seizure disorder, unstable spinal
    cord compression, superior vena cava syndrome, extensive interstitial bilateral lung
    disease on high resolution computed tomography (HRCT) scan or any other condition that
    would limit compliance with study requirements.

    - Patients may not require concomitant use of known strong CYP3A inhibitors (e.g.,
    itraconazole, telithromycin, clarithromycin, protease inhibitors boosted with
    ritonavir or cobicistat, indinavir, saquinavir, nelfinavir, boceprevir, telaprevir) or
    moderate CYP3A inhibitors (e.g., ciprofloxacin, erythromycin, diltiazem, fluconazole,
    verapamil). The required washout period prior to starting study treatment is 2 weeks.

    - Patients may not require concomitant use of known strong (e.g., phenobarbital,
    enzalutamide, phenytoin, rifampicin, rifabutin, rifapentine, carbamazepine, nevirapine
    and St John's Wort) or moderate CYP3A inducers (e.g. bosentan, efavirenz, modafinil).
    The required washout period prior to starting study treatment is 5 weeks for
    enzalutamide or phenobarbital and 3 weeks for other agents.

    Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

    Eligibility last updated 6/9/23. Questions regarding updates should be directed to the study team contact.

    Drug
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    Mayo Clinic — Rochester, MN

    Post-approval Registry Study to Evaluate the Continued Safety and Probable Benefit of the MID-C System for 5 Years Post-Implantation in Adolescent Idiopathic Scoliosis (AIS)

    Study to Evaluate the Continued Safety and Probable Benefit of the MID-C System for 5 Years Post-Implantation in Adolescent Idiopathic Scoliosis (AIS)

    Annalise Larson
    All
    10 years to 25 years old
    This study is NOT accepting healthy volunteers
    2020-101934-P01-RST
    20-002525
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    Inclusion Criteria:

    • Adolescent Idiopathic Scoliosis classified as Lenke Type 1 or Type 5 curves.
    • Cobb angle between 40-60 degrees (inclusive).
    • Flexible curve that that reduces to ≤ 30 degrees on lateral side bending radiographs or as evident by traction x-ray.
    • Kyphosis angles of ≤ 55 degrees measured from T5 to T12.
    • Appropriate candidate for posterior surgical approach.
    • Patient has good general health.
    • Patient has no known hypersensitivity or allergies to titanium.
    • Patient’s guardian signs a written informed consent form (ICF).


    Exclusion Criteria:

    • Any type of non-idiopathic scoliosis.
    • Any main thoracic deformity that includes vertebral levels and cranial including to T2.
    • Known history of existing malignancy, or any systemic or local infection.
    • Spinal cord abnormalities that require treatment.
    • Known neurological deficit (defined as motor grade < 5/5).
    • Known poor bone quality defined as T score -1.5 or less.
    • For female patient, pregnancy.
    • Previous spine surgery.
    • Active systemic disease, such as AIDS, HIV, or active infection.
    • Active infection or the skin is compromised at the surgical site.
    • Systemic disease that would affect the Patient’s welfare or overall outcome of the study.

     

    Juvenile scoliosis, Scoliosis
    Adolescent idiopathic scoliosis, Musculoskeletal system, Scoliosis surgery, Spinal deformity correction surgery
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    Mayo Clinic — Rochester, MN

    Post-approval Registry Study to Evaluate the Continued Safety and Probable Benefit of the MID-C System for 5 Years Post-Implantation in Adolescent Idiopathic Scoliosis (AIS)

    Study to Evaluate the Continued Safety and Probable Benefit of the MID-C System for 5 Years Post-Implantation in Adolescent Idiopathic Scoliosis (AIS)

    Annalise Larson
    All
    10 years to 25 years old
    This study is NOT accepting healthy volunteers
    2020-101934-P01-MPMC
    20-002525
    Show full eligibility criteria
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    Inclusion Criteria:

    • Adolescent Idiopathic Scoliosis classified as Lenke Type 1 or Type 5 curves.
    • Cobb angle between 40-60 degrees (inclusive).
    • Flexible curve that that reduces to ≤ 30 degrees on lateral side bending radiographs or as evident by traction x-ray.
    • Kyphosis angles of ≤ 55 degrees measured from T5 to T12.
    • Appropriate candidate for posterior surgical approach.
    • Patient has good general health.
    • Patient has no known hypersensitivity or allergies to titanium.
    • Patient’s guardian signs a written informed consent form (ICF).


    Exclusion Criteria:

    • Any type of non-idiopathic scoliosis.
    • Any main thoracic deformity that includes vertebral levels and cranial including to T2.
    • Known history of existing malignancy, or any systemic or local infection.
    • Spinal cord abnormalities that require treatment.
    • Known neurological deficit (defined as motor grade < 5/5).
    • Known poor bone quality defined as T score -1.5 or less.
    • For female patient, pregnancy.
    • Previous spine surgery.
    • Active systemic disease, such as AIDS, HIV, or active infection.
    • Active infection or the skin is compromised at the surgical site.
    • Systemic disease that would affect the Patient’s welfare or overall outcome of the study.

     

    Juvenile scoliosis, Scoliosis
    Adolescent idiopathic scoliosis, Musculoskeletal system, Scoliosis surgery, Spinal deformity correction surgery
    I'm interested
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    Location Contacts
    Mayo Clinic Square — Minneapolis, MN