Health Studies MN
Treatment of ARDS With Instilled T3 (ARDS+T3)
Study objective: To determine the safety and tolerability of Thyroid Hormone (T3) delivery into the lungs of Acute Respiratory Distress Syndrome (ARDS) patients, and to measure the effect of T3 on extravascular lung water in ARDS patients.
Ronald Reilkoff
rreilkof@umn.edu
All
18 Years and over
Phase 1/Phase 2
NCT04115514
STUDY00007410
Inclusion Criteria:
Clinical diagnosis of ARDS:
• Chest x-ray: bilateral pulmonary infiltrates
• Hypoxemia: PaO2:FIO2 ratio <200
• Volume status: wedge and CVP<18 Main inclusion criteria:
• Adults (≥18 years of age), non-pregnant
• On mechanical ventilatory support
Exclusion Criteria:
1. Inadequate medical history for determining inclusion/exclusion criteria, as determined
by the Principal Investigator and/or Sub-Investigators.
2. Unlikely to complete the protocol with clinic follow-up after discharge, as determined
by the Principal Investigator and/or Sub-Investigators or hospice status.
3. Active drug/alcohol use with positive drug screen or alcohol level on admission.
4. Prior history of thyroid cancer or hyperthyroidism, per thorough patient/family
interviews, review of past medical history, medication list, laboratory test.
5. Prior history of cardiovascular disease including:
1. Hypertensive crisis in the past 3 months (systolic >200, or diastolic >120 mmHg),
2. Sustained ventricular arrhythmia in the past 3 months (duration > 30 seconds)
3. Coronary artery disease (documented >50% occlusion in any coronary vessel)
4. Cardiac-related angina pectoris (> 2 episodes in the past 3 months)
5. Myocardial infarction with ischemia on ECG (i.e., new ST-elevation/depression of
>1mm in contiguous leads), or positive cardiac enzymes (Ratio of CK-MB: Total CK
> 3.5).
6. Peripheral vascular disease (documented >50% occlusion in any peripheral vessel).
7. Moderate or severe ischemic/non-ischemic cardiomyopathy (documented ejection
fraction < 40%).
8. Decompensated or symptomatic heart failure (i.e., hospitalized for CHF
exacerbation, or a change in CHF medications within two weeks prior)
6. Currently pregnant or breastfeeding.
7. Currently taking tricyclic antidepressants, glycosides, ketamine, or vasopressors with
ongoing evidence of myocardial ischemia.
8. Known allergy to study drug.
Drug: Liothyronine Sodium (T3) (modified formulation)
ARDS, Human, Lung, Wet, Thyroid, Pulmonary Edema, Lung Inflammation
Confocal Laser Endomicroscopy as an Imaging Biomarker for the Diagnosis of Pancreatic Cystic Lesions (CLIMB)
This study will evaluate EUS-nCLE (Endoscopic ultrasound needle-based Confocal Laser Endomicroscopy) as an imaging biomarker and PCL (pancreatic cystic lesion) fluid DNA analysis as a molecular biomarker for the management of PCLs. Each of the objectives will be assessed by: Utilizing EUS-nCLE alone, utilizing EUS-nCLE with cyst fluid molecular markers, optimal combination of clinical features, imaging features, cyst fluid analysis, EUS-nCLE, and cyst fluid molecular markers. The primary objectives will be to: distinguish between mucinous and non-mucinous PCLs, distinguish mucinous PCLs that have advanced neoplasia (high-grade dysplasia or cancer) from those with low-grade dysplasia, and distinguish individual distinct types of PCLs.
All
18 Years and over
NCT03492151
Inclusion Criteria:
• Patient age 18 years or older
• All patients referred for EUS-FNA of accessible PCL where surgery is contemplated
• Minimum cyst size should be ≥ 2.0 cm as determined by prior cross-sectional imaging studies
Exclusion Criteria:
• Unable to obtain informed consent
• Unable to tolerate the procedure
• Women with known pregnancy at time of procedure
• Patient age less than 18 years
• Bleeding diathesis
• Known allergy to fluorescein
• Prior pancreatic cancer
• Prior pancreatic surgery
Pancreatic Cyst
Confocal laser endomicroscopy
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
NCT04293185
STUDY00006923
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 severe VOEs in the 24 months prior to informed consent as defined below. A protocol-defined severe VOE is: (a) an event of acute priapism: defined as a sustained, unwanted painful erection lasting more than 2 hours and requiring care at a medical facility (with or without hospitalization) or (b) an event that requires a ≥ 24-hour hospital or emergency room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment.
• 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 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) (e.g. TCD velocity >200 cm/sec) requiring 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-lymphotrophic virus-1 (HTLV-1) or -2 (HTLV-2), 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 in the bone marrow that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
Genetic: bb1111
Sickle Cell Disease
The Up-LIFT Study of Non-Invasive ARC Therapy for Spinal Cord Injury (Up-LIFT)
To provide pilot evidence to the FDA for the LIFT System (a transcutaneous spinal cord stimulator) that the system is safe, effective, and evaluate potential benefits. This is a multisite industry sponsored study (GTX Medical, Inc., Lexington, MA).
All
22 Years to 75 Years old
N/A
NCT04697472
Key
Inclusion Criteria:
Subjects must meet all the following criteria:
1. At least 22 years old and no older than 75 years old at the time of enrollment
2. Non-progressive cervical spinal cord injury from C2-C8 inclusive
3. American Spinal Injury Association (ASIA) Impairment Scale (AIS) classification B, C,
or D
4. Indicated for upper extremity training procedures by subject's treating physician or a
physical therapist
5. Minimum 12 months post-injury
6. Capable of providing informed consent
Key Exclusion Criteria:
Subjects must not meet any of the following criteria:
1. Has uncontrolled cardiopulmonary disease or cardiac symptoms as determined by the
Investigator
2. Has any unstable or significant medical condition that is likely to interfere with
study procedures or likely to confound study endpoint evaluations like severe
neuropathic pain, depression, mood disorders or other cognitive disorders
3. Has been diagnosed with autonomic dysreflexia that is severe, unstable, and
uncontrolled
4. Requires ventilator support
5. Has an autoimmune etiology of spinal cord dysfunction/injury
6. Spasms that limit the ability of the subjects to participate in the study training as
determined by the Investigator
7. Breakdown in skin area that will come into contact with electrodes
8. Has any active implanted medical device
9. Pregnant, planning to become pregnant or currently breastfeeding
10. Concurrent participation in another drug or device trial that may interfere with this
study
11. In the opinion of the investigators, the study is not safe or appropriate for the
participant
Device: LIFT System
Chronic Spinal Cord Injury
SCI, Tetraplegia, ARC Therapy
JSP191 Antibody Targeting Conditioning in SCID Patients
Phase 1: To evaluate the safety and tolerability of JSP191 and to determine Phase 2 doses of JSP191 as a conditioning agent prior to allogeneic hematopoietic cell transplantation (HCT) in two populations of subjects with severe combined immunodeficiency (SCID): • SCID subjects with history of prior allogeneic HCT but with poor graft function • SCID subjects who are HCT-naïve Phase 2: • To evaluate the efficacy of JSP191 conditioning to enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism • To evaluate the efficacy of JSP191 conditioning to enable immune reconstitution determined by the production of naïve T cells
Christen Ebens
ebens012@umn.edu
All
3 Months and over
Phase 1/Phase 2
NCT02963064
STUDY00010559
Key
Inclusion Criteria:
All patient groups must have:
1. Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but
not limited to the following subtypes:
1. T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient
2. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
3. T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient OR Variant
SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular
dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase
deficiency may be included after consultation with the medical monitor.
2. Patients with human leukocyte antigen (HLA) matched related or unrelated donors
3. Adequate end organ function as defined in study protocol
Key Exclusion Criteria:
1. Patients with any acute or uncontrolled infections
2. Patients receiving any other investigational agents, or concurrent biological,
chemotherapy, or radiation therapy
3. Patients with active malignancies
4. Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for
GVHD
Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)
SCID
Immunodeficiency, Pediatric, SCID, Bone Marrow Transplantation, GVHD, Stem Cells, Chimerism, Transplant, BMT
Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
The overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
Claudio Brunstein, MD
bruns072@umn.edu
All
12 Years and over
Phase 3
NCT04260698
STUDY00010454
Inclusion Criteria:
• Patients must be at least 12 years of age
• Applicable disease criteria
• Patients must have one or two partially HLA-matched CBUs
• Back-up stem cell source
• Sufficient physiological reserves
• Females of childbearing potential agree to use appropriate method of contraception
• Signed written informed consent
Exclusion Criteria:
• Extensive bone marrow fibrosis
• Donor specific anti-HLA antibodies
• Pregnancy
• Medically unsuitable for transplant
Biological: omidubicel
Hematological Malignancies
Rare CFTR Mutation Cell Collection Protocol (RARE) (RARE)
We are doing the RARE study to learn more about Cystic Fibrosis (CF). CF is caused by mutations in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). In people with CF, the CFTR does not function correctly. Medications are being developed to help the CFTR function better, but those medications mostly benefit people with common CFTR mutations. There are more than 1,900 mutations of the CF gene. Some of these mutations are rare and found only in a few people. The goal of this research study is to collect specimens (blood, nasal cells, rectal cells) from people with rare CFTR mutations. Another purpose of this study is to create induced pluripotent stem cells or iPS cells. “Pluripotent” stem cells are cells that can be changed into almost any cell type of the body (such as lung or intestine). They can be kept alive and stored indefinitely. There are different kinds of pluripotent stem cells. Inducted pluripotent stem cells can be created from many different kinds of specimens (such as blood, nasal cells, rectal cells). This is different from embryonic stem cells, which can only be derived from embryos. The specimens collected during this study and iPS cells created from them will be stored for use in future research to learn more about CF and study the effect of new medications. This could identify new medications that may help people with rare CFTR mutations.
Joanne Billings
billi001@umn.edu
All
12 Years and over
NCT03161808
1702M07621
Inclusion Criteria:
• Male or female ≥ 12 years of age at time of consent
• Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with CF and one or more of the following criteria (1. Sweat chloride ≥ 60 milliequivalents/Liter (mEq/L) by quantitative pilocarpineiontophoresis test (QPIT) OR upon permission of the RARE Investigator- Sponsors, 2. Two well-characterized mutations in the cystic fibrosis transmembrane conductive regulator (CFTR) gene, 3.Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproterenol of lessthan -6.6 mV)
• Confirmed genotype of the current recruitment focus for certain target rare mutations. The initial recruitment focus will be CF patients who are homozygous for pre-mature stop codons. Operations Memos will detail any future current genotype targets.
• Written informed consent (and assent when applicable) obtained from participant or participant's legal representative and ability to comply with the requirements of the study.
• Willing to travel (if needed) to a regional study site for cell collection.
Exclusion Criteria:
1. Presence of a medical condition, abnormality, or laboratory value(s) that in the
opinion of the onsite principal investigator and/or collaborating gastroenterologist
may compromise the quality of the data or place the subject at significant risk by
undergoing the research related biopsy, including:
Significantly diseased distal rectal/GI tissue that could place the participant at
risk by participating in the study (as judged by the collaborating gastroenterologist,
such as significant hemorrhoids, vascular abnormalities, colonic infection, radiation
injury or history of radiation therapy to the rectum, prostate and/or pelvic area)
Any of the following abnormal lab values at the study visit:
i. Platelets < 50 x 103/µL ii. Hemoglobin < 10 gm/dL iii. Hematocrit < 30% iv. WBC >
20 x 103/µL v. Neutropenia (ANC < 1.5 x 103/µL) vi. Lymphopenia (absolute lymphocyte
count < 1.5 x 103/µL) vii. PT/INR > 1.5 viii. Other bleeding diathesis
2. Positive pregnancy test (for female of childbearing potential) at the study visit.
3. Breastfeeding (if patient opts to use sedation).
4. Current use of drugs with significant risks of compromising immunity (e.g. oral
steroid use >20 mg/day) for >14 days prior to the rectal biopsy.
5. History of organ transplant.
6. Use of oral anticoagulant medications (e.g., chronic anticoagulant therapy such as
warfarin or platelet inactivators such as aspirin) within seven days prior to rectal
biopsy.
7. Unable or unwilling to withhold use of oral anticoagulant medications (e.g., chronic
anticoagulant therapy such as warfarin or platelet inactivators such as aspirin)
within 7 days after rectal biopsy.
Cystic Fibrosis
Androgen Deprivation Therapy (ADT) and Pembrolizumab for Advanced Stage Androgen Receptor-positive Salivary Gland Carcinoma
Manish Patel
patel069@umn.edu
All
18 Years and over
Phase 2
NCT03942653
STUDY00004710
Inclusion Criteria:
• Written informed consent and HIPAA authorization for release of personal health information. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
• Age ≥ 18 years at the time of consent.
• Locally advanced, recurrent, or metastatic salivary gland carcinoma that is not amenable to curative surgery or radiation
• ECOG Performance Status of 0 or 1 within 28 days prior to registration.
• Local, pathologic testing of androgen receptor-positive salivary gland carcinoma will be performed as standard of care. Archival tissue must be available for central confirmation of androgen receptor-positive disease and for correlative studies. AR positivity will be defined according to IHC staining of tumor tissue with at least 20% of tumor staining positive with moderate intensity (1+ or greater).
• Measurable disease according to RECIST v1.1 for solid tumors within 28 days prior to registration.
• For patients who have been treated with prior therapy, patients must have documented progression of disease on their prior therapy for entry into the study.
• Patients with prior chemotherapy, radiation, or surgery as part of curative intent therapy are allowed. Any number of prior lines of systemic therapy is permitted for entry into this study so long as prior therapy did not include anti-androgen therapy or immune checkpoint blockade.
• If prior cancer treatment, the subject must have recovered from toxic effects of prior cancer treatment (other than alopecia) to ≤ Grade 1.
• Adequate organ function as defined below; all screening labs to be obtained within 28 days prior to registration.
• Absolute neutrophil count (ANC) ≥1500/µL
• Platelets ≥75,000/µL
• Hemoglobin ≥8.0 g/dL or ≥5 mmol/L
• Creatinine (Cr) OR Measured or calculated creatinine clearance (GFR can also be used in place of Cr or creatinine clearance) ≤1.5 × ULN OR ≥30 mL/min for participant with creatinine levels >1.5 × institutional ULN
• Total bilirubin ≤1.5 ×ULN OR direct bilirubin ≤ULN for participants with total bilirubin levels >1.5 × ULN
• AST (SGOT) and ALT (SGPT) ≤2.5 × ULN (≤5 × ULN for participants with liver metastases) o International normalized ratio (INR) OR prothrombin time (PT) & aPTT ≤1.5 × ULN unless participant is receiving anticoagulant therapy as long as PT or aPTT is within therapeutic range of intended use of anticoagulants
• A male participant must agree to use contraception during the treatment period and for at least 8 months after the last dose of study treatment and refrain from donating sperm during this period.
• Females of childbearing potential must have a negative serum pregnancy test within 72 hours prior to registration. NOTE: Females are considered of child bearing potential unless they are surgically sterile (have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are naturally postmenopausal for at least 12 consecutive months
• Females of childbearing potential and males with partners of childbearing potential must be willing to abstain from heterosexual activity or to use a highly effect form of contraception from the time of informed consent until 8 months after treatment discontinuation.
• As determined by the enrolling physician or protocol designee, ability of the subject to understand and comply with study procedures for the entire length of the study
Exclusion Criteria:
• Women of childbearing age with a positive serum pregnancy test within 72 hours prior to study registration.
• Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, CTLA-4, OX40, CD137).
• Has received prior androgen deprivation therapy including orchiectomy, gonadotropin-releasing hormone (GnRH) agonists/antagonists, androgen receptor blocker, abiraterone, or enzalutamide.
• Has received prior systemic anti-cancer therapy including investigational agents within 14 days prior to registration.
• Has received prior palliative radiotherapy within 7 days of start of study treatment. Participants must have recovered from all radiation-related toxicities and require less than 10mg of prednisone (or equivalent corticosteroid) daily.
• Has received a live vaccine within 28 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella (MMR), varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (eg, FluMist®) are live attenuated vaccines and are not allowed.
• Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 7 days prior to the first dose of study drug.
• Has a known additional malignancy that is progressing or has required active treatment within the past 2 years. Note: Participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (e.g. breast ductal carcinoma in situ, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
• Has known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, i.e. without evidence of progression for at least 14 days by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable, and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment.
• Has ≥Grade 3 hypersensitivity to pembrolizumab and/or any of its excipients.
• Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg, levothyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
• Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis.
• Has an active infection requiring systemic therapy.
• Has a known history of Human Immunodeficiency Virus (HIV).
• Has a known history of active TB (Bacillus Tuberculosis).
• Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
• Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 120 days after the last dose of trial treatment.
Drug: Goserelin Acetate, Drug: Pembrolizumab
Salivary Gland Carcinoma
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
This an extension study assessing the use of migalastat (AT1001) in pediatric populations. AT1001, under the trade name Galafold, is approved for use in the US in adults, but not children. The parent study is approved by the IRB under STUDY00006216.
Chester Whitley, MD, PhD
whitley@umn.edu
All
12 Years to 17 Years old
Phase 3
NCT04049760
STUDY00009760
Inclusion Criteria:
• Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
Exclusion Criteria:
• Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
• Has advanced kidney disease requiring dialysis or kidney transplantation
• History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
• Has received any gene therapy at any time or anticipates starting gene therapy during the study period
• Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
• Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
• Subject is treated or has been treated with any investigational/experimental drug, biologic or device within 30 days before screening
• Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
• Pregnant or breast-feeding
• Otherwise unsuitable for the study in the opinion of the investigator
Drug: migalastat HCl 150 mg
Fabry Disease
Lysosomal storage disease, migalastat
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (Imagine-1)
This study is a prospective multi-cohort, open-label, dose-escalation assessment of the safety and efficayc of PBGM01, an AAVHu68, intra-cisternal magna delivered gene therapy for the treatment of GM1 gangliosidosis in infants 1-24 months of age.
All
4 Months to 36 Months old
Phase 1/Phase 2
NCT04713475
Inclusion Criteria:
• All Patients: Documented GM1 gangliosidosis diagnosis based on genotyping confirming 2 mutations in the GLB1 gene and documented deficiency of beta-galactosidase enzyme by laboratory testing
• Age: 4 to 36 months (first cohort will be 12-36 months) Subjects:
• Early onset infantile (Type 1): Subjects who have signs and/or symptoms of GM1 gangliosidosis that started at or before 6 months of age and have specific developmental milestones remaining
• Late onset infantile (Type 2a): Subjects who have signs and/or symptoms of GM1 gangliosidosis that started between 6 and 18 months of age and have specific developmental milestones remaining
Exclusion Criteria:
1. Any clinically significant neurocognitive deficit not attributable to GM1
gangliosidosis or any other condition that may, in the opinion of the investigator,
confound interpretation of study results.
2. If a subject had an acute illness requiring hospitalization within 30 days of
enrollment, the history must be discussed with the sponsor's medical monitor before
allowing the subject to be enrolled.
3. History of ventilation assisted respiratory support or a need for tracheostomy as a
result of their disease.
4. Intractable seizure or uncontrolled epilepsy defined as having had an episode of
status epilepticus, or seizures requiring hospitalization within 30 days prior to
dosing of PBGM01.
5. Any contraindication to the ICM administration procedure, including contraindications
to fluoroscopic imaging and anesthesia or any condition that would increase the risk
of adverse outcomes from the ICM procedure including, but not limited to, the presence
of a space occupying lesion causing mass effects or signs of increased intracranial
pressure, space occupying lesion in the posterior fossa or foramen magnum, aberrant
vascular anatomy such as a large midline posterior inferior cerebellar artery,
aberrant venous anatomy such as a large cerebellar vein or occipital sinus, or
congenital anatomical abnormalities such as a Chiari malformation.
6. Any contraindication to MRI or lumbar puncture (LP).
7. Prior gene therapy.
8. Use of miglustat within 48 hours prior to dosing of PBGM01. The use of miglustat is
prohibited throughout the study.
9. Use of enzyme replacement therapy or other investigational therapy within 5 half-lives
prior to dosing of PBGM01. The use of enzyme replacement is prohibited throughout the
study.
10. Receipt of a vaccine within 14 days of dosing.
11. Estimate glomerular filtration rate (eGFR) <30 mL/minute based on creatinine
12. Coagulopathy (INR > 1.5) or activated partial thromboplastin time [aPTT] > 40 seconds
13. Thrombocytopenia (platelet count < 100,000 per μL.
14. AST or ALT > 3 times the upper limit of normal (ULN) or total bilirubin > 1.5x ULN
15. Cardiomyopathy (screening troponin level above the ULN).
16. Peripheral neuropathy
17. Medical conditions or laboratory or vital sign abnormalities that would increase risk
of complications from intra-cisterna magna injection, anesthesia, fluoroscopy, LP,
and/or MRI including temperature over 38°C, oxygen saturation below 95% on room air or
baseline oxygen requirement, heart rate or respiratory rate abnormal for age of the
subject, abnormal blood pressure for age, or evidence of infection.
18. Any condition (e.g., history of any disease, evidence of any current disease, any
finding upon physical examination, or any laboratory abnormality) that, in the opinion
of the investigator, would put the subject at undue risk during the administration
procedure or would interfere with evaluation of PBGM01 or interpretation of subject
safety or study results.
Biological: PBGM01
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Infantile, Late Infantile, Rare disease, Lysosomal storage disease
Study of BGB-A1217 in Combination With Tislelizumab in Advanced Solid Tumors
Manish Patel
patel069@umn.edu
All
18 Years and over
Phase 1
NCT04047862
STUDY00011977
Key
Inclusion Criteria:
Phase 1 Key Inclusion Criteria
1. Has Eastern Cooperative Oncology Group (ECOG) Performance Status ≤1.
2. ≥ 1 measurable lesion per RECIST v1.1.
3. Has adequate organ function.
4. phase 1- Patients with histologically or cytologically confirmed advanced, metastatic,
unresectable solid tumors who have previously received standard systemic therapy or
for which treatment is not available, not tolerated or refused.
Phase 1b Key Inclusion Criteria
1. Signed informed consent form (ICF) and able to comply with study requirements.
2. Age ≥ 18 years (or the legal age of consent) at the time the ICF is signed.
3. Histologically or cytologically confirmed tumor types in the following disease
cohorts:
Cohort 1: stage IV squamous NSCLC Cohort 2: stage IV non-squamous NSCLC Cohort 3:
stage IV squamous or non-squamous NSCLC with PD-L1 positive. Cohort 4: extensive-stage
SCLC Cohort 5: stage IIIB, IIIC or IV NSCLC Cohort 6: stage IV ESCC Cohort 7: stage IV
EAC Cohort 8: recurrent or metastatic HNSCC incurable by local therapies Cohort 9:
stage IV G/GEJ adenocarcinoma.
4. ECOG Performance Status ≤ 1
5. Adequate organ function
6. Willing to use highly effective method of birth control
Phase 1 Key Exclusion Criteria:
1. Active brain or leptomeningeal metastasis.
2. Active autoimmune diseases or history of autoimmune diseases that may relapse.
3. With severe chronic or active infections requiring systemic antibacterial, antifungal
or antiviral therapy, including tuberculosis infection, etc. (antiviral therapy is
permitted for patients with hepatocellular carcinoma).
4. Concurrent participation in another therapeutic clinical trial.
5. Received prior therapies targeting TIGIT.
Phase 1b Key Exclusion Criteria:
1. Patients with any prior therapy for recurrent/metastatic disease.
2. Non-squamous NSCLC patients with sensitizing epidermal growth factor receptor (EGFR)
mutation, anaplastic lymphoma kinase (ALK) fusion, and c-ros oncogene 1 (ROS1) fusion.
3. Gastric cancer patients with squamous or with positive HER2 expression.
4. Prior therapy with any drug specifically targeting T-cell co-stimulation or checkpoint
pathways. (anti-PD(L)1 exception for Cohort 5).
5. Active leptomeningeal disease or uncontrolled brain metastasis.
6. Active autoimmune diseases or history of autoimmune diseases that may relapse.
7. With severe chronic or active infections requiring systemic antibacterial, antifungal
or antiviral therapy, including tuberculosis infection, etc. (antiviral therapy is
permitted for patients with hepatocellular carcinoma).
8. Concurrent participation in another therapeutic clinical study.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: BGB-A1217, Drug: Tislelizumab, Drug: Pemetrexed, Drug: Paclitaxel, Drug: Nab paclitaxel, Drug: Carboplatin, Drug: Cisplatin, Drug: Etoposide, Drug: 5fluorouracil, Drug: Oxaliplatin, Drug: Capecitabine
Locally Advanced and Metastatic Solid Tumors
BGB-A1217, Anti-TIGIT antibody, Tislelizumab, anti-PD-1
STABILITY 2: Anterior Cruciate Ligament Reconstruction +/- Lateral Tenodesis With Patellar vs Quad Tendon (STABILITY 2)
Jeffrey Macalena
maca0049@umn.edu
All
14 Years to 25 Years old
N/A
NCT03935750
STUDY00010820
Inclusion Criteria:
• Age 14-25,
• An ACL-deficient knee,
• Skeletal maturity (i.e. closed epiphyseal growth plates on standard knee radiographs),
• At least two of the following: participate in a competitive pivoting sport; have a pivot shift of grade 2 or greater; have generalized ligamentous laxity (Beighton score of ≥4) and/or genu recurvatum >10 degrees.
Exclusion Criteria:
• Previous ACLR on either knee,
• Partial ACL injury (defined as one bundle ACL tear requiring reconstruction/augmentation of the torn bundle with no surgery required for the intact bundle),
• Multiple ligament injury (two or more ligaments requiring surgery),
• Symptomatic articular cartilage defect requiring treatment other than debridement,
• >3 degrees of asymmetric varus,
• Inflammatory arthropathy,
• Inability to provide consent,
• Pregnancy at baseline.
Procedure: Anterior cruciate ligament reconstruction (ACLR), Procedure: Lateral extra-articular tenodesis (LET)
Anterior Cruciate Ligament Injury, Anterior Cruciate Ligament Reconstruction, Joint Instability
Lateral extra-articular tenodesis, Autografting, Allografting, Bone-Patellar Tendon-Bone Grafting
A Study of JNJ-64304500 as Add-on Therapy in Participants With Active Crohn's Disease (DUET)
All
18 Years to 65 Years old
Phase 2
NCT04655807
Inclusion Criteria:
• Have confirmed clinical diagnosis of Crohn's disease or fistulizing Crohn's disease of at least 3 months' duration
• Initiated standard of care (SOC) biologic therapy for at least 12 uninterrupted weeks (including the induction dose) prior to Week 0 and agree to continue to maintain their SOC biologic with no change in dose level or interruption for the duration of the study. Adalimumab (including HUMIRA or an equivalent biosimilar which could include: HULIO, HYRIMOZ, IMRALDI, or AMGEVITA) at maintenance dose of 40 milligram (mg) subcutaneous (SC) every 2 weeks (q2w) plus minus (+ -) 4 days or Ustekinumab at maintenance dose of 90 mg SC every 8 weeks (q8w) +
•7 days
• Have active Crohn's disease (CD), with a baseline crohn's disease activity index (CDAI) score of greater than or equal to (>=) 180 but less than or equal to (<=) 400
• Participant with a family history of colorectal cancer, personal history of increased colorectal cancer risk, age greater than (>) 50 years, or other known risk factor must be up-to-date on colorectal cancer surveillance
• Participant who has had extensive colitis for >=8 years, or disease limited to the left side of the colon for >=12 years, must either have had a colonoscopy to assess for the presence of dysplasia within 1 year before the first administration of study agent or a colonoscopy to assess for the presence of malignancy at the screening visit, with no evidence of malignancy
• A woman of childbearing potential must have a negative highly sensitive serum (beta- human chorionic gonadotropin [beta-hCG]) pregnancy test result at screening and a negative urine pregnancy test result at Week 0 and throughout the study
Exclusion Criteria:
• Has complications of Crohn's disease as defined in study protocol
• Currently has or is suspected to have an abscess
• Concomitant or previous medical therapies received: has previously demonstrated suboptimal response, loss of response, or intolerance to more than 2 approved advanced therapies
• Concomitant or previous medical therapies received: corticosteroids and 5-aminosalicylic acid (5-ASA) compounds at unstable or above recommended doses are not permitted. Individuals receiving stable doses (oral corticosteroids at a prednisone-equivalent dose at or below 20 mg/day, or 6 mg/day of budesonide, 2.5 mg/day beclomethasone dipropionate, or at or below 5-ASA doses of 1.5 gram (g)/day) or if individuals have been discontinued, for at least 2 weeks before start of first study intervention (Week 0), are permitted
• Concomitant or previous medical therapies received: has received any of the following prescribed medications or therapies within the specified period or has plans to initiate throughout the study: conventional immunomodulators (that is , azathioprine [AZA], 6-mercaptopurine [6 MP], or methotrexate [MTX]) within 4 weeks of first dose of study intervention; oral immunomodulatory agents (example, 6-thioguanine [6-TG], cyclosporine, tacrolimus, sirolimus, or mycophenolate mofetil, tofacitinib and other Janus kinase [JAK] inhibitors [including investigational JAK inhibitors]) less than (<) 6 weeks or within 5 half-lives of agent before first dose of SOC biologic, whichever is longer; all other immunomodulatory biologic agents (including investigational biologics) received within 12 weeks or within 5 half-lives of first dose of SOC biologic, whichever is longer
• Infections or predisposition to infections criteria: has a stool culture or other examination positive for an enteric pathogen, including clostridium difficile toxin, in the last 4 months unless a repeat examination is negative and there are no signs of ongoing infection with that pathogen
• Has a transplanted organ (with exception of a corneal transplant that needs to have occurred > 12 weeks before screening)
Drug: JNJ-64304500, Drug: Placebo, Drug: Adalimumab, Drug: Ustekinumab
Crohn Disease
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in parent clinical studies. Lenti-D Drug Product is defined as an autologous CD34+ cell-enriched population that contains cells transduced with Lenti-D lentiviral vector encoding the human adrenoleukodystrophy protein. In parent studies, male subjects with CALD are infused on a single occasion with Lenti-D Drug Product, and then followed for 24 (±1) month for safety and efficacy. The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) recommend long-term follow-up for subjects treated with gene therapy drug products to monitor for selected adverse events (AEs), as well as durability of clinical response. Therefore, after subjects have completed the parent clinical studies, they will be asked to participate in a long-term follow-up Study LTF-304, in which they will be followed every 6 months through 5 years post-drug product infusion, and then annually through 15 years post-drug product infusion. Safety evaluations will include documentation of drug product-related AEs, all serious adverse events (SAEs) regardless of attribution to the drug product, CALD-related ≥Grade 2 AEs, and integration site analysis for the detection of clonal dominance through 15 years post drug product infusion, as well as archiving for RCL testing through 5 years post‑drug product infusion. Efficacy evaluations will include CALD disease-specific assessments, primarily major functional disabilities and brain MRI, with additional exploratory assessments for change in Loes score, Loes pattern, neurologic function score (NFS), very long chain fatty acids (VLCFA), intelligence quotient (IQ), and health related quality of life (HRQoL) assessment. To monitor pharmacodynamics, vector copy number in peripheral blood (PB VCN; vector copies per diploid genome [c/dg]) and transgenic protein expression of adrenoleukodystrophy protein (ALDP) in peripheral blood will be measured at designated study visits. There is no designated Data Monitoring Committee (DMC) for Study LTF-304; however, the review of safety data for this study, including AEs, SAEs and relevant laboratory values, may be performed by the DMC convened for the parent study in which the subject(s) originally participated.
Paul Orchard
orcha001@umn.edu
Male
Not specified
NCT02698579
STUDY00005330
Inclusion Criteria:
• Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
• Have received Lenti-D Drug Product in a parent clinical study
• Able to comply with study requirements
Exclusion Criteria:
• There are no exclusion criteria for this Study
Genetic: Lenti-D
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation
Define natural history, validate patient reported outcome and share knowledge on congenital disorders of glycosylation. We will recruit and enroll patients with CDG in this study evaluating clinical variation and natural history when a patient is being seen as part of routine clinical care.
Kyriakie Sarafoglou
saraf010@umn.edu
All
Not specified
NCT04199000
STUDY00009013
Inclusion Criteria:
• Patients diagnosed with congenital disorders of glycosylation based on genetic confirmatory testing
Exclusion Criteria:
• Patients without congenital disorders of glycosylation
Congenital Disorders of Glycosylation
CDG, CDDG, Congenital Disorders of Glycosylation, Congenital Disorders of Deglycosylation, ALG1, ALG3, ALG6, ALG12, ALG13, COG6, DPAGT1, DPM1, EDEM3, MAN1B1, MPDU1, MPI, NGLY1, PGAP3, PGM1, PIGA, PIGG, PIGN, PIGS, PIGT, PMM2, SLC35A2, SLC35C1, SLC39A8, SRD5A3, SSR4, FUT8, GALNT2, MAN2B2, VMA21
Cusatuzumab in Combination With Background Therapy for the Treatment of Participants With Acute Myeloid Leukemia (ELEVATE)
All
18 Years and over
Phase 1
NCT04150887
Inclusion Criteria:
• Diagnosis of acute myeloid leukemia (AML) according to World Health Organization 2016 criteria . Participants with acute promyelocytic leukemia (APL) are not eligible
• Must be ineligible for intensive chemotherapy
• De novo or secondary AML
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
• Previously untreated AML except: emergency leukapheresis, hydroxyurea, and/or 1 dose 1-2 gram per meter square (g/m^2) cytarabine during the Screening Phase to control hyperleukocytosis. These treatments must be discontinued greater than or equal to (>=) 24 hours prior to start of study drug. Empiric all trans retinoic acid (ATRA) treatment for presumed acute promyelocytic leukemia (APL) is permitted but APL must be ruled out and ATRA must be discontinued >=24 hours prior to the start of study drug
• Contraceptive use by men or women should be consistent with local regulations regarding the use of contraceptive methods for participants participating in clinical studies
Exclusion Criteria:
• Leukemic involvement of the central nervous system
• Eligible for an allogeneic hematopoietic stem cell transplantation at study entry
• Received a live, attenuated vaccine within 4 weeks prior to initiation of study drug
• A history of human immunodeficiency virus (HIV) antibody positive or tests positive for HIV if tested at screening
• Known allergies, hypersensitivity, or intolerance to cusatuzumab, venetoclax, azacitidine, or their excipients (example: mannitol, an excipient of azacitidine)
Drug: Cusatuzumab, Drug: Azacitidine, Drug: Venetoclax
Leukemia, Myeloid, Acute
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
This is a prospective, multinational, multicenter, open-label, non-randomized, first-in human Phase 1/2a, dose-escalation study to evaluate the safety, tolerability, and exploratory efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease. Participants will be treated in sequential, dose-level cohorts. The number of participants in each cohort will be determined by levels of circulating GAA, safety, and immunogenicity evaluations. Data from participants treated early in the study will enable potential adaptation of the dosing regimen for an optional additional cohort(s) or for an expanded cohort (or cohorts) at selected dose levels.
Chester Whitley, MD, PhD
whitley@umn.edu
All
18 Years and over
Phase 1/Phase 2
NCT04093349
STUDY00008038
Inclusion Criteria:
• Provide written informed consent;
• Males and Females ≥18 years of age with late-onset Pompe disease;
• Received ERT for at least the previous 24 months
• Have clinically moderate, late-onset Pompe disease characteristics;
• Agree to use reliable contraception.
Exclusion Criteria:
• Active hepatitis B and/or C;
• Significant underlying liver disease;
• Human immunodeficiency virus (HIV) infection;
• Prior hypersensitivity to rhGAA;
• Pre-existing anti-AAV neutralizing antibody titers;
• High titer antibody responses to rhGAA;
• Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
• Received any prior vector or gene transfer agent;
• Active malignancy (except non-melanoma skin cancer);
• History of liver cancer;
• Pregnant or nursing women;
• Any evidence of active infection at the time of SPK-3006 infusion.
Genetic: SPK-3006
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Advanced Materials Science in XLIF Study (AMS in XLIF)
This study is a prospective, non-concurrent, multicenter study to compare the clinical and radiographic outcomes of smooth Polyetheretherketone (PEEK), 3D-printed titanium, and Porous PEEK interbody implants when used with cancellous allograft chips with Bone Marrow Aspirate (BMA) or cellular allograft in subjects who undergo lumbar lateral interbody fusion at one or two levels. Patients will be followed up to 24 months post-surgery. Fusion rates and clinical outcomes of the 3 groups will be evaluated.
Jonathan Sembrano
sembr001@umn.edu
All
18 Years to 80 Years old
NCT03649490
STUDY00004973
Inclusion Criteria:
1. Male and female patients who are 18-80 years of age;
2. Planned interbody fusion surgery, including at least one level of extreme lateral
interbody fusion (XLIF) prior to enrollment in the research at one or two consecutive
lumbar levels for degenerative disc disease, including those with up to Grade 2
spondylolisthesis, with one of the following NuVasive, Inc. interbody implants:
1. Coroent® XL PEEK interbody implant; or
2. Modulus® 3D-printed titanium interbody implant; or
3. Cohere® XLIF Porous PEEKTM interbody implant.
If a transforaminal lumbar interbody fusion or anterior lumbar interbody fusion (TLIF
or ALIF) is planned adjacent to the XLIF level, the same NuVasive, Inc. interbody
implant material type must be used as determined by the implant enrollment schedule.
For example:
1. Smooth PEEK: Coroent XL PEEK (XLIF) + Coroent L (TLIF) or Coroent XLR (ALIF) or
Brigade (ALIF)
2. 3D-printed titanium: Modulus XLIF + Modulus TLIF or Modulus ALIF
3. Porous PEEK: Cohere XLIF + Coalesce TLIF (currently no ALIF option)
3. The planned procedure must include placement of bilateral posterior screw fixation
with or without intrafacet fusion using autograft (with or without the assigned
allograft used at the XLIF level(s)) at the treated level(s). Direct posterior
decompression at the index interbody fusion level(s) is acceptable.
4. Preoperative coronal Cobb angle of < 10°;
5. Able to undergo surgery based on physical exam, medical history, and surgeon judgment;
6. Understands the conditions of enrollment and willing to sign an informed consent to
participate in the evaluation.
Exclusion Criteria:
1. Use of BMP, synthetic bone graft substitutes, allografts, or any other graft material
in the interbody or intrafacet spaces other than those under study;
2. Posterior grafting other than the allowed intrafacet fusion at the treated level(s);
3. Revision of prior fusion at treated level(s) (adjacent level interbody fusion is
acceptable);
4. XLIF procedure that requires or results in the release of the anterior longitudinal
ligament or posterior osteotomy;
5. Preoperative coronal Cobb angle of ≥ 10°;
6. Procedures performed with XLIF interbody implants with integrated vertebral body
screw(s);
7. Active smoking six (6) weeks prior to surgery;
8. Systemic or local infection (active or latent);
9. Diseases that significantly inhibit bone healing (e.g., prior diagnosis of
osteoporosis, metabolic bone disease, uncontrolled diabetes, dialysis dependent renal
failure, symptomatic liver disease);
10. Rheumatoid arthritis or other autoimmune disease that, in the option of the
investigator, would interfere with bone healing and/or fusion;
11. Treatment with pharmaceuticals interfering with calcium metabolism;
12. Undergoing chemotherapy or radiation treatment or chronic use of steroids (defined as
more than 6 weeks of steroid use within 12 months of surgery or anytime
postoperatively, other than episodic use or inhaled corticosteroids);
13. Use of bone stimulators postoperatively;
14. Non-ambulatory, wheelchair-bound;
15. Involvement in active litigation relating to the spine (worker's compensation claim is
allowed if it is not contested);
16. Significant general illness (e.g., HIV, active metastatic cancer of any type,
uncontrolled diabetes, dialysis dependent renal failure, symptomatic liver disease);
17. Spinal metastases or active spinal tumor malignancy;
18. Immunocompromised or is being treated with immunosuppressive agents;
19. Pregnant, or plans to become pregnant during the study;
20. Mental or physical condition that would limit the ability to comply with study
requirements;
21. Prisoners;
22. Participating in another clinical study that would confound study data.
Degenerative Disc Disease, Spondylolisthesis
Study of NKTR 255 in Combination With Cetuximab in Solid Tumors
This study is a Phase 1b (Dose Escalation) / 2 (Dose Expansion), open-label, multicenter dose escalation and dose expansion study in patients with relapsed or refractory (R/R) head and neck squamous cell carcinoma (HNSCC) or colorectal carcinoma (CRC). The intervention is FDA-approved cetuximab combined with an investigational drug, NKTR-255. Patients will receive a loading dose of cetuximab alone, followed 7 days later by the first combination treatment of cetuximab and NKTR-255 on Cycle 1 Day 1. Thereafter, NKTR-255 will be given in 21-day cycles in combination with weekly IV cetuximab. After determination of the recommended Phase 2 dose (RP2D) of NKTR-255 in combination with cetuximab, this dose of NKTR-255 will be further studied in patients with HNSCC (Cohort A) and CRC (Cohort B) in Phase 2 of the study. Patients will remain on treatment until meeting one of the criteria for discontinuation.
Manish Patel
patel069@umn.edu
All
18 Years and over
Phase 1/Phase 2
NCT04616196
STUDY00011716
Key
• Histologically confirmed diagnosis of a locally advanced or metastatic HNSCC or CRC.
• Life expectancy > 12 weeks as determined by the Investigator.
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
• Measurable disease per RECIST 1.1. HNSCC: Progression on any first or second line platinum-based chemotherapy and/or anti-PD-1 or programmed death-ligand 1 antibody. CRC: Patients must have received or were intolerant to at least 2 prior cancer therapy regimens administered for metastatic disease. CRC: Patients with microsatellite instability-high (MSI-H) or mismatched repair disease (dMMR) tumors must have been exposed to checkpoint inhibitors such as anti-PD-(L)1 or anticytotoxic T-lymphocyte-associated protein (CTLA)-4 antibody. Key
• Use of an investigational agent or an investigational device within 28 days before administration of first dose of study drug(s)
• Prior surgery or radiotherapy within 14 days of initiating study drug(s)
• Evidence of clinically significant interstitial lung disease or active, noninfectious pneumonitis; active infection requiring systemic therapy within 7 days prior to dosing
• Patients who have been previously treated with IL-2 or IL-15
• Contraindication to or unable to receive cetuximab
Inclusion Criteria:
• Histologically confirmed diagnosis of a locally advanced or metastatic HNSCC or CRC.
• Life expectancy > 12 weeks as determined by the Investigator.
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
• Measurable disease per RECIST 1.1. HNSCC: Progression on any first or second line platinum-based chemotherapy and/or anti-PD-1 or programmed death-ligand 1 antibody. CRC: Patients must have received or were intolerant to at least 2 prior cancer therapy regimens administered for metastatic disease. CRC: Patients with microsatellite instability-high (MSI-H) or mismatched repair disease (dMMR) tumors must have been exposed to checkpoint inhibitors such as anti-PD-(L)1 or anticytotoxic T-lymphocyte-associated protein (CTLA)-4 antibody. Key
Exclusion Criteria:
• Use of an investigational agent or an investigational device within 28 days before administration of first dose of study drug(s)
• Prior surgery or radiotherapy within 14 days of initiating study drug(s)
• Evidence of clinically significant interstitial lung disease or active, noninfectious pneumonitis; active infection requiring systemic therapy within 7 days prior to dosing
• Patients who have been previously treated with IL-2 or IL-15
• Contraindication to or unable to receive cetuximab
Drug: NKTR-255, Drug: NKTR-255
Head and Neck Squamous Cell Carcinoma (HNSCC), Colorectal Cancer (CRC)
HNSCC, CRC, NKTR-255, Cetuximab, Phase I Clinic, Head and Neck Squamous Cell Carcinoma, Colorectal Cancer, cSCC, Cutaneous Squamous Cell Carcinoma, ASCC, Anal Squamous Cell Carcinoma, Cervical Cancer, Erbitux®
A Sequenced Strategy for Improving Outcomes in People With Knee Osteoarthritis Pain (SKOAP)
There is an urgent public health need to reduce our reliance on opioids for effective long-term pain management, particularly in knee osteoarthritis (KOA). This effectiveness trial will compare recommended treatments to reduce pain and functional limitations in KOA and identify clinical and patient-level factors associated with treatment response. These results will lead to improved patient selection for treatment and inform evidence based guidelines by offering well-tested, effective, non-opioid alternatives.
All
18 Years to 90 Years old
Phase 3
NCT04504812
Inclusion Criteria:
• Meets American College of Rheumatology Classification criteria for knee osteoarthritis
Exclusion Criteria:
• Any inability to complete study procedures, including, but not limited to low English language literacy.
• Inability to access the internet on a daily basis
• Unstable medical condition that presents as an absolute or relative contraindication for participation (e.g., unstable angina, poorly controlled diabetes mellitus, end stage renal failure, automated implantable cardioverter-defibrillator that cannot be disabled before RFA).
• Severe untreated bleeding disorder (anticoagulants may be continued during phase II treatments in most patients)
• Severe vision or hearing impairment or serious cognitive impairment that could interfere with consent or outcome assessment
• Poorly controlled serious psychiatric condition
• Active substance abuse
• Scheduled joint replacement on the affected knee
• History of unilateral total knee arthroplasty (TKA) with complaints of KOA pain limited to the operated knee
• Ulcers or an open wound in the region of the index knee
Drug: Duloxetine, Combination Product: Intra-Articular Injection, Procedure: Nerve Procedure with long acting blocks, Procedure: Nerve Procedure with nerve ablation, Behavioral: Pain Coping Skills Training, Other: Best Practices
Knee Osteoarthrosis
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)
All
2 Years and over
Phase 2
NCT04732429
Inclusion Criteria:
• Confirmed diagnosis of symptomatic PA or MMA (Mutase)
• Ages ≥ 2 years old.
• History of Inadequate metabolic control while receiving standard of care (SoC).
• Plasma MCA concentration > 3x upper limit of normal of the reference range at screening.
• Stable supplementation dose of carnitine for at least 1 week prior to the entry in the study.
Exclusion Criteria:
• Moderate-to-severely impaired cardiac function with LVEF < 45% by ECHO.
• Clinically significant arrhythmia by Holter monitor.
• QTcF > 450 msec
• Moderate to severe chronic kidney disease with estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2.
• Exposure to any investigational therapy, apart for a COVID-19 vaccine, within the past 6 months prior to study entry.
• Exposure to gene therapy for PA or MMA at any time prior to study entry.
• History of organ transplantation (Part A and B only)
• History of severe allergic or anaphylactic reactions to any of the components of HST5040.
Drug: HST5040, Drug: Placebo
Methylmalonic Acidemia, Propionic Acidemia
Methylmalonic Acidemia, Propionic Acidemia, Organic Acidemia, Inborn errors of metabolism, PCCA, PCCB, Propionyl-coenzyme A carboxylase, MMUT, Methylmalonyl-CoA mutase, Metabolic disease, Genetic disease, HemoShear
Evaluation of Pain Before and After Removal of Non-obstructive Kidney Stones (ENORC)
To prospectively determine if the removal of non-obstructing renal calculi can reduce or eliminate a participant’s pain and/or improve their quality of life. We hypothesize that the removal of non-obstructing renal calculi will decrease or eliminate the participant’s pain and will improve their quality of life.
All
18 Years and over
N/A
NCT03657667
Inclusion Criteria:
• Patients with renal colic and non-obstructing renal calculi. No stone greater than 10 mm in longest diameter
• All other causes of pain have been eliminated (by clinical judgment; if the cause of pain is in doubt: assessment by a family doctor or medical specialist will be obtained)
• Patients older than 18 years old
• Moderate to severe pain (> or = 5 on BPI pain scale: pain at its worst in the last 24hrs)
Exclusion Criteria:
• Patient's with anatomic abnormalities (calyceal diverticulum)
• Ureteral calculi
• Nephrocalcinosis
• RTA, medullary sponge kidney, sarcoidosis
• Hydronephrosis or hydrocalycosis
• Minimal pain (<5 on BPI pain scale: pain at its worst in the last 24 hrs)
Procedure: Ureteroscopy
Kidney Stone
Renal Stone, Nephrolithiasis, Painful stone in kidney, Kidney stone pain
A Phase I/II Study of Nivolumab, Ipilimumab and Plinabulin in Patients With Recurrent Small Cell Lung Cancer
This is an open-label Phase I/II study, with a dose escalation part (Phase I) and a 2-arm randomized part (Phase II), in patients with recurrent small cell lung cancer. In the Phase I part, patients will receive plinabulin at escalating doses in combination with nivolumab and ipilimumab. In the Phase II part, approximately 40 patients will be randomized in a 1:1 ratio to receive either nivolumab + ipilimumab (Arm NI) or the triple combination of plinabulin (at the recommended phase 2 dose) + nivolumab + ipilimumab (Arm PNI). Patients will continue treatment until disease progression, development of unacceptable toxicity or one of the protocol-defined reasons for treatment discontinuation occurs.
Naomi Fujioka
fujio002@umn.edu
All
18 Years and over
Phase 1/Phase 2
NCT03575793
STUDY00004593
Inclusion Criteria:
The patients must satisfy all of the following inclusion/exclusion criteria in order to be
eligible for the study:
• Must have signed and dated written informed consent form in accordance with regulatory and institutional guidelines.
• Males and females aged >18 years at time of consent.
• Histological or cytological confirmed extensive-stage SCLC
• Patients who progressed after at least 1 platinum-based chemotherapy regimen. Patients with platinum resistance (defined as recurrence or progression of disease within 90 days of completion of the platinum-based regimen) are eligible. For phase II, patients also must have been treated with at least one prior line of PD-1/PD-L1 therapy.
• Measurable disease according to RECIST v1.1 (Section 8) obtained by imaging within 28 days prior to study registration.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 within 14 days before registration and minimum life expectancy of at least 12 weeks.
• Treatment to be initiated at least 2 weeks since last dose of prior systemic anticancer therapy (chemotherapy, radiation, and/or surgery.
• Recovery to grade 1 of any clinically significant toxicity (excluding alopecia, grade 2 fatigue, vitiligo, endocrinopathies on stable replacement therapy) prior to initiation of study drugs.
• Female patients of childbearing potential have a negative pregnancy test at baseline. Females of childbearing potential are defined as sexually mature women without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, women who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti estrogens, or ovarian suppression.
• Women of childbearing potential (i.e., menstruating women) must have a negative urine pregnancy test (positive urine tests are to be confirmed by serum test) documented within 14 days of study registration and within the 24-hour period prior to the first dose of study drug.
• Sexually active women of childbearing potential enrolled in the study must agree to use 2 forms of accepted methods of contraception during the course of the study and for 23 weeks after their last dose of study drug. Effective birth control includes (a) intrauterine device plus 1 barrier method; (b) on stable doses of hormonal contraception for at least 3 months (e.g., oral, injectable, implant, transdermal) plus one barrier method; (c) 2 barrier methods. Effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm); or (d) a vasectomized partner.
• For male patients who are sexually active and who are partners of premenopausal women: agreement to use 2 forms of contraception as in criterion 9b above during the treatment period and for 31 weeks after the last dose of study drug.
• Adequate laboratory values.
• Absolute neutrophil count ≥1,000/µL
• Platelet count ≥100,000/µL
• Hemoglobin ≥9.0 g/dL
• Total bilirubin ≤1.5 x upper limit of normal (ULN) or ≤3 x ULN for subjects with Gilbert's disease
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3.0 x ULN (≤5 x ULN if evidence of hepatic involvement by malignant disease)
• Creatinine ≤ 1.5 x ULN or estimated glomerular filtration rate (eGFR) ≥40 mL/min/1.73m2
• Lipase and Amylase ≤1.5 x ULN. Subjects with Lipase >1.5 x ULN may enroll if there are neither clinical nor radiographic signs of a pancreatitis. Exclusion Criteria Patients with any of the following will be excluded from participation in the study.
• Active interstitial lung disease (ILD) or pneumonitis or a history of ILD or pneumonitis requiring treatment with steroids. Prior history of radiation pneumonitis is allowed if pneumonitis was restricted to the field of radiation.
• History of ileus or other significant gastrointestinal disorder known to increase the risk of ileus or chronic bowel hypomotility
• Pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 23 weeks (female) or 31 weeks (male) after the last dose of study drug.
• Must not have received CTLA-4 targeted therapy previously
• Treatment with any investigational agent within 28 days prior to registration for protocol therapy. Vaccination for SARS-CoV-2 is allowed as well as any therapy as required for the treatment of active COVID 19 infection.
• Known active symptomatic central nervous system (CNS) metastases and/or carcinomatous meningitis. Patients with neurological symptoms must undergo a head computed tomography (CT) scan or brain magnetic resonance imaging (MRI) to exclude brain metastasis. Patients whose brain metastases have been treated may participate provided there is no evidence of progression for at least 2 weeks after CNS-directed treatment, as ascertained by clinical examination or brain imaging.
• Known history of human immunodeficiency virus (HIV) or active hepatitis B (by surface antigen expression or polymerase chain reaction [PCR]) or active hepatitis C (by PCR) infection. NOTE: HIV testing is not required; Hepatitis B and C testing are required at screening.
• Diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to study registration.
• Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e., with use of disease modifying agents, corticosteroids, or immunosuppressive drugs) or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Vitiligo, alopecia, hypothyroidism only requiring hormone replacement, psoriasis not requiring systemic treatment, celiac disease controlled by diet alone or conditions not expected to recur in the absence of an external trigger are permitted.
• A condition requiring systemic treatment with corticosteroids (>10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days prior to administration of study drugs.
• History of psychiatric illness or social situations that would limit compliance with study requirements. Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the patient's participation for the full duration of the trial, or is not in the best interest of the patient to participate, in the opinion of the treating investigator.
• Prior malignancies (except non-melanoma skin cancers, and the following in situ cancers: bladder, gastric, colon, endometrial, cervical/dysplasia, melanoma, or breast) unless a complete remission was achieved at least 2 years prior to study entry.
• Documented history of a cerebral vascular event (stroke or transient ischemic attack), unstable angina, myocardial infarction, or cardiac symptoms consistent with New York Heart Association (NYHA) Class III-IV within 6 months prior to their first dose of study drugs.
• Evidence of ongoing inadequately controlled hypertension (defined as baseline systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg).
• Any active grade 3 or higher viral, bacterial, or fungal infection within 2 weeks of the first dose of the study drugs. Routine antimicrobial prophylaxis is permitted.
Drug: Nivolumab, Drug: Plinabulin, Drug: Ipilimumab
Lung Cancer, SCLC
A Prospective Observational Study of TPIAT (POST)
Aim 1: To determine (1a) whether patient and disease characteristics are associated with favorable pain and health-related quality-of-life outcomes (HRQOL) after TPIAT; (1b) the optimal timing of the TPIAT intervention to resolve pain and improve HRQOL; and (1c) in a subset of patients, the impact of central sensitization on pain resolution. Aim 2: To determine (2a) whether patient and disease characteristics are associated with favorable glycemic outcomes from the IAT procedure; and (2b) the optimal timing of TPIAT to obtain post-surgical insulin independence. Aim 3: To determine the cost-effectiveness of TPIAT.
Melena Bellin
bell0130@umn.edu
All
Not specified
NCT03260387
1512M81750
Inclusion Criteria:
1. Any patient with chronic or recurrent acute pancreatitis undergoing total or completion
pancreatectomy with islet autotransplantation at a participating center.
Exclusion Criteria:
1. Partial pancreatectomy
2. TPIAT performed for a diagnosis other than chronic or recurrent acute pancreatitis
(for example benign or malignant pancreatic tumor)
Procedure: TPIAT
Pancreatitis, Chronic, Pancreatectomy, Hyperglycemia, Pancreatitis
pediatric, total pancreatectomy, acute pancreatitis, chronic pancreatitis, islet transplant, islet autotransplant
Tumor-Agnostic Precision Immuno-Oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study
TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in patients with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are TMB-high as identified by a validated NGS assay.
Emil Lou
emil-lou@umn.edu
All
Not specified
Phase 2
NCT04589845
STUDY00010440
Inclusion Criteria:
• Histologically or cytologically confirmed diagnosis of advanced and unresectable or metastatic solid malignancy
• Measurable disease as defined by Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1), Response Assessment in Neuro-Oncology (RANO) criteria, or International Neuroblastoma Response Criteria (INRC)
• Performance status as follows: Participantss aged >= 18 years: Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2; Participantss aged 16 to < 18 years: Karnofsky score >= 50%; Participants aged < 16 years: Lansky score >= 50%
• For participants aged >= 18 and <18 years: adequate hematologic and end-organ function
• Disease progression on prior treatment, or previously untreated disease with no available acceptable treatment
• Adequate recovery from most recent systemic or local treatment for cancer
• Life expectancy >= 8 weeks
• Ability to comply with the study protocol, in the investigator's judgment
• For female participants of childbearing potential: Negative serum pregnancy test <= 14 days prior to initiating study treatment; agreement to remain abstinent or use single or combined contraception methods that result in a failure rate of < 1% per year for the period defined in the cohort-specific inclusion criteria; and agreement to refrain from donating eggs during the same period
• For male participants: Willingness to remain abstinent or use acceptable methods of contraception as defined in the cohort-specific inclusion criteria
• In addition to the general inclusion criteria above, participants must meet all of the cohort-specific inclusion criteria for the respective cohort
Exclusion Criteria:
• Current participation or enrollment in another therapeutic clinical trial
• Any anticancer treatment within 2 weeks or 5 half-lives prior to start of study treatment
• Whole brain radiotherapy within 14 days prior to start of study treatment
• Stereotactic radiosurgery within 7 days prior to start of study treatment
• Pregnant or breastfeeding, or intending to become pregnant during the study
• History of or concurrent serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study or confounds the ability to interpret data from the study
• Incomplete recovery from any surgery prior to the start of study treatment that would interfere with the determination of safety or efficacy of study treatment
• Significant cardiovascular disease, such as New York Heart Association cardiac disease (Class II or higher), myocardial infarction, or cerebrovascular accident within 3 months prior to enrollment, unstable arrhythmias, or unstable angina
• History of another active cancer within 5 years prior to screening that may interfere with the determination of safety or efficacy of study treatment with respect to the qualifying solid tumor malignancy
• In addition to the general exclusion criteria above, in order to be enrolled in a treatment cohort of the study, participants must not meet any of the cohort-specific exclusion criteria
Drug: Entrectinib, Drug: Entrectinib, Drug: Alectinib, Drug: Atezolizumab, Drug: Ipatasertib, Drug: Trastuzumab emtansine, Drug: Idasanutlin, Drug: Inavolisib, Drug: Belvarafenib, Drug: Pralsetinib
Solid Tumors
Phase I Clinic
Pediatric cGVHD Symptom Scale
To develop a psychometrically valid Pediatric cGVHD Symptom Scale (PCSS) and a companion parent-proxy measure as counterparts to the Lee cGVHD Symptom Scale.
Margaret MacMillan, MD
macmi002@umn.edu
All
5 Years and over
NCT04044365
STUDY00008469
• INCLUSION CRITERIA: Pediatric Subject
Inclusion Criteria:
• Children aged 5 to 17 years old, who have undergone prior allogeneic stem cell transplant
• Clinical diagnosis of cGVHD
• Currently receiving systemic treatment for cGVHD (including phototherapies), or has had systemic therapy for cGVHD tapered to discontinuation within the past 12 months
• No evidence of malignant disease relapse including molecular relapse and minimal residual disease. Patients with mixed chimerism are eligible to participate
• Parent or guardian ability and willingness to sign a written informed consent document
• Subjects must be able to comprehend and speak the English language
• Subjects may participate in both Project 1 and Project 2 of the study. Participation in Project 1 is not required in order to be eligible to participate in Project 2.
• Parent or Guardian Proxy Inclusion Criteria
• Parent or guardian of participating subject
• Must be willing and able to provide informed consent. Parent or guardian (proxy) must be able to comprehend and speak the English language EXCLUSOIN CRITERIA:
• Patients who completed systemic treatment more than 3 months prior to enrollment or are receiving topical therapy only.
• Patients may be excluded from this study if in the judgment of the Principal or Associate Investigator, the subject is too ill, or subject s cognitive ability would compromise their ability to participate in study related procedures.
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation (HSCT), Allogeneic Stem Cell Transplant, Phototherapies, Graft Vs Host Disease, Natural History
A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)
Primary objective, Part A (dose escalation): To assess the safety of escalating doses of CTX110 in subjects with relapsed or refractory B cell malignancies to determine the recommended Part B dose Primary objective, Part B (cohort expansion): To assess the efficacy of CTX110 in subjects with relapsed or refractory B cell malignancies, as measured by objective response rate (ORR)
Joseph Maakaron
maaka001@umn.edu
All
18 Years and over
Phase 1
NCT04035434
STUDY00010648
Key
Inclusion Criteria:
1. For NHL patients: Age ≥18 years. For B cell ALL patients: age ≥18 years to ≤70 years
2. Refractory or relapsed non-Hodgkin lymphoma, as evidenced by 2 or more lines of prior
therapy, or histologically confirmed B cell ALL, refractory or relapsed.
3. Eastern Cooperative Oncology Group performance status 0 or 1.
4. Adequate renal, liver, cardiac and pulmonary organ function
5. Female subjects of childbearing potential and male subjects must agree to use
acceptable method(s) of contraception from enrollment through at least 12 months after
CTX110 infusion.
6. Agree to participate in an additional long-term follow-up study after completion of
this study.
Key Exclusion Criteria:
1. Treatment with any gene therapy or genetically modified cell therapy, including CAR T
cells.
2. For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic
HSCT within 6 months, and/or any evidence of GvHD.
3. History of central nervous system (CNS) involvement by malignancy
4. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia,
cerebellar disease, or any autoimmune disease with CNS involvement.
5. Presence of bacterial, viral, or fungal infection that is uncontrolled or requires IV
anti-infectives.
6. Active HIV, hepatitis B virus or hepatitis C virus infection.
7. Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma,
adequately resected and in situ carcinoma of cervix, or a previous malignancy that was
completely resected and has been in remission for ≥5 years.
8. For NHL patients: Use of systemic anti-tumor therapy or investigational agent within
14 days or 5 half-lives, whichever is longer, of enrollment. For B cell ALL patients:
Use of systemic antitumor therapy within 7 days of enrollment.
9. Primary immunodeficiency disorder or active autoimmune disease requiring steroids
and/or other immunosuppressive therapy.
10. Women who are pregnant or breastfeeding.
Biological: CTX110
B-cell Malignancy, Non-Hodgkin Lymphoma, B-cell Lymphoma, Adult B Cell ALL
CAR T, Non-Hodgkin Lymphoma, NHL, Lymphoma, Allogeneic, Leukemia
Validation of Bladder Health Instrument for Evaluation in Women (VIEW)
Female
18 Years and over
NCT04016298
Inclusion Criteria:
• Community dwelling
• Age ≥18 years old
• Female sex assigned at birth
• Fluent in written and spoken English
• Able to read and provide informed consent
Exclusion Criteria:
• Institutional living arrangement, e.g., skilled nursing, long term care or rehabilitation center
• Physical or mental condition that would prohibit self-administration of questionnaire either electronically or using paper and pencil (e.g., dementia/cognitive impairment/blindness/severe arthritis).
Lower Urinary Tract Symptoms
Bladder Health, LUTS, PLUS, VIEW
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS) (POPS or POP02)
The majority of drugs administered to children are used off label, and PK studies to define appropriate dosing are lacking across pediatric age groups and special populations of children. Challenges associated with clinical trials in children limit the ability to conduct PK and dosing trials in this population. Studies capitalizing on standard-of-care procedures have proven successful in characterizing the PK of drugs used in children. The purpose of this study is to characterize the PK of understudied drugs administered to children per SOC as prescribed by their treating provider. This study will serve as a tool to better understand drug exposure in children receiving drugs per SOC. The data collected through this initiative will provide valuable PK and dosing information for drugs in different pediatric age groups as well as special populations of children, such as premature infants, critically ill children receiving ECMO or CRRT, children with Down syndrome and children with obesity, for which dosing may vary due to altered PK. In addition, the data collected in this study will serve as preliminary data to design and plan the best and most efficacious BPCA trials, proof-of-concept studies associated with biomarkers, and data to support applications for extramural funding. All of the drugs studied in this protocol are used as standard of care in children and are approved in adults. There will be multiple INDs held by the core study Principal Investigator: Danny Benjamin, MD, PhD (IND Sponsor) Kiser-Arena Distinguished Professor of Pediatrics, Duke University Faculty Associate Director, Duke Clinical Research Institute PO Box 17969 Durham NC 27715 Phone: 919-668-8295 Fax: 919-681-9457 danny.benjamin@duke.edu The Funding Sponsor is The National Institute of Child Health and Human Development (NICHD) NOTE: We will be participating in the COVID 19 arm of this study, which includes 6 drugs of interest (DOI). All other arms are on hold currently; and focus has been placed on the COVID 19 arm. However, in the future we may be interested in participating in other DOIs. The details of this arm of the study will be provided at the end of this document. (See Appendix P, pages 82-86 of main protocol)
Catherine Bendel
bende001@umn.edu
All
up to 20 Years old
NCT04278404
STUDY00009884
Inclusion Criteria:
1. Participant is < 21 years of age and
1. is receiving understudied drugs of interest (DOIs) per standard of care (SOC) as
prescribed by their treating provider OR
2. is NOT receiving one or more of the study drugs of interest but is SARS-CoV-2
positive within 60 days prior to enrollment
2. Parent/ Legal Guardian/ Adult Participant can understand the consent process
and is willing to provide informed consent/HIPAA
Exclusion Criteria:
1. Participant has a known pregnancy
For participants receiving one or more of the study drugs of interest at the
time of enrollment, DOI administration or PK sampling:
(Refer to DOI specific appendices for details on enrollment cohort
specifications)
2. Has had intermittent dialysis within previous 24 hours
3. Has had a kidney transplant within previous 30 days
4. Has had a liver transplant within previous 1 year
5. Has had a stem cell transplant within previous 1 year
6. Has had therapeutic hypothermia within previous 24 hours
7. Has had plasmapheresis within the previous 24 hours
8. Has a Ventricular Assist Device
9. Has any condition which would make the participant, in the opinion of the
investigator, unsuitable for the study
Drug: The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension, Pain, Hyperphosphatemia, Primary Hyperaldosteronism, Edema, Hypokalemia, Heart Failure, Hemophilia, Menorrhagia, Insomnia, Pneumonia, Skin Infection, Arrythmia, Asthma in Children, Bronchopulmonary Dysplasia, Adrenal Insufficiency, Fibrinolysis, Hemorrhage, Attention Deficit Hyperactivity Disorder, Multisystem Inflammatory Syndrome in Children (MIS-C), Kawasaki Disease, Coagulation Disorder, Down Syndrome
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Peter Karachunski
karac001@umn.edu
All
Not specified
NCT04174157
STUDY00008659
Inclusion Criteria:
• Patients with SMA, genetically confirmed on or after 24 May 2018.
• Appropriate consent/assent has been obtained for participation in the registry
Exclusion Criteria:
•Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA. Note: Patients that are participating in a Compassionate Use Program (CUP) for AVXS-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of genetic confirmation of SMA.
Other: Prospective observational registry, Drug: Zolgensma
Spinal Muscular Atrophy (SMA)