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.
• 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.)
Fecal Microbiome Transplant National Registry
Fecal Microbiome Transplant National Registry
- Recipient
- Ability to give informed consent
- Receiving FMT or other gut-related microbiota product within 90 days after providing consent
- Access to internet and/or telephone
- Donor
- Ability to give informed consent
- Providing stool sample for FMT
- Incarceration
Fecal Microbiota Transplant National Registry (FMT)
Fecal Microbiota Transplant National Registry
-
Recipient Inclusion Criteria
- Ability to give informed consent
- Receiving FMT or other gut-related microbiota product within 90 days after providing consent
- Access to internet and/or telephone
-
Donor Inclusion
- Ability to give informed consent
- Providing stool sample for FMT
- Incarceration
COllaborative, National QUality and Efficacy Registry for Tracking Disease Progression in Systemic Sclerosis (Scleroderma) Patients (CONQUER) (CONQUER)
A Registry to Track Scleroderma Progression
- Patients 18 and older, inclusive; AND
- Patients who meet the 2013 ACR/EULAR classification criteria for systemic sclerosis; AND
- < 5 years from onset of first non-RP symptom attributed to systemic sclerosis.
- Patients under 18 years of age.
- Patients with cognitive impairment that will interfere with conducting the study. Study staff should determine if the patient, at that moment, has the cognitive capacity to understand what the study entails, assent to participate, and complete the assessments. If there is question as to whether a patient may meet this criterion, RCs/RAs should defer to the site PI trained on the study to help determine; OR
- Patients who are non-English Speaking. The patient must be able to proficiently read, speak, and understand English in order to be eligible.
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
• Patients with SMA, genetically confirmed on or after 24 May 2018.
• Appropriate consent/assent has been obtained for participation in the registry
•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.
PIANO: Pregnancy in Inflammatory Bowel Disease and Neonatal Outcomes: Long-term Outcomes of In-Utero Biologic and Thiopurine Exposure (PIANO)
A Multicenter National Prospective Study of Pregnancy and Neonatal Outcomes in Women With Inflammatory Bowel Disease
- ≥ 18 years of age.
- Female patients with confirmed IBD diagnoses who are pregnant and taking immunosuppressants and/or biologic therapy.
- Pregnant female patients younger than 18 years of age.
- Confirmed multiple gestation.
Eligibility last updated 10/18/21. Questions regarding updates should be directed to the study team contact.
The NODE-303 Study: Multi-Centre, Multi-National,Open Label, Safety Study of Etripamil Nasal Spray for Patients With Paroxysmal Supraventricular Tachycardia. (NODE-303)
Multi-Centre, Multi-National, Open Label, Safety Study of Etripamil Nasal Spray for Patients with Paroxysmal Supraventricular Tachycardia
- Has been diagnosed with PSVT by a medical professional, and reports having at least one previous episode of PSVT. For clarity, PSVT refers to episodic SVT that includes the AV node as a critical part of reentrant circuit.
- Is at least 18 years of age.
- Signed NODE-303 written informed consent.
- Women of child-bearing potential must be willing to use at least 1 form of contraception during the trial and must be willing to discontinue from the study should they become or plan to become pregnant. Postmenopausal females are defined as having amenorrhea for at least 12 months prior to Screening without an alternative medical cause.
- Willing and able to comply with study procedures.
- Patients with only a history of atrial arrhythmia that does not involve the atrioventricular (AV) node as part of the tachycardia circuit (e.g., atrial fibrillation, atrial flutter, intra-atrial tachycardia) are not eligible. Patients with a history of these tachycardias who are also diagnosed with PSVT are eligible.
- History of allergic reaction to verapamil.
- Current therapy with digoxin, or any Class I or III antiarrhythmic drug. Patients may be eligible if these drugs are stopped at least five half-lives before the administration of etripamil NS. The only exception is oral amiodarone which must be stopped 30 days before enrollment.
- History or evidence of ventricular pre-excitation; e.g., delta waves, Wolff-Parkinson-White syndrome.
- History or evidence of a second- or third-degree AV block.
- History or evidence of severe ventricular arrhythmia (e.g., torsades de pointes, ventricular fibrillation, or sustained ventricular tachycardia).
- Symptoms of congestive heart failure New York Heart Association Class II to IV.
- SBP < 90 mmHg at Screening, Baseline or any Follow-up Visit.
- Severe symptoms of hypotension experienced during PSVT episodes.
- Significant physical or psychiatric condition including alcoholism or drug abuse, which, in the opinion of the Investigator, could jeopardize the safety of the patient, or impede the patient’s capacity to follow the study procedures.
- History of syncope due to an arrhythmic etiology at any time, or history in last 5 years of unexplained syncope.
- Is pregnant or breastfeeding.
- Previously enrolled in a clinical trial for etripamil and received study drug or participation in any clinical trial for other investigational products or medical devices within 30 days of Screening.
- History of ACS or stroke within 6 months of Screening.
- Evidence of renal dysfunction as determined by an estimated glomerular filtration rate assessed at the Screening Visit as follows:
- < 60mL/min/1.73m^2 for patients < 60 years of age;
- < 40mL/min/1.73m^2 for patients ≥ 60 and < 70 years of age;
- < 35mL/min/1.73m^2 for patients ≥ 70 years of age.
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.
• 0 through 55 years of age
• Adequate graft available
• Adequate organ function
• Eligible Diseases:
• Mucopolysaccharidosis Disorders:
• MPS IH (Hurler syndrome)
• MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
• MPS VI (Maroteaux-Lamy syndrome)
• MPS VII (Sly syndrome)
• Glycoprotein Metabolic Disorders:
• Alpha mannosidosis
• Fucosidosis
• Aspartylglucosaminuria
• Sphingolipidoses and Recessive Leukodystrophies:
• Globoid cell leukodystrophy
• Metachromatic leukodystrophy
• Niemann-Pick B patients (sphingomyelin deficiency)
• Niemann-Pick C subtype 2
• Peroxisomal Disorders:
• Adrenoleukodystrophy with cerebral involvement
• Zellweger syndrome
• Neonatal Adrenoleukodystrophy
• Infantile Refsum disease
• Acyl-CoA-Oxidase Deficiency
• D-Bifunctional enzyme deficiency
• Multifunctional enzyme deficiency
• Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
• Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
• Severe Osteopetrosis (OP)
• Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
• Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.
• Voluntary written consent
• Pregnancy
•menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
• Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
• Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
University of Minnesota Transplant Registry
• Received a transplanted organ at the University of Minnesota
• Living donor who donates an organ at the University of Minnesota
• Did not receive a transplant at the University of Minnesota
• Did not donate an organ at the University of Minnesota
(EpiAcc Registry) Percutaneous Epicardial Access Registry (EpiAcc Registry)
Percutaneous Epicardial Access Registry
- All patients 18 years of age and older undergoing (or who have previously undergone) EpiAcc as part of their standard clinical care for an electrophysiology procedure. Patients will not undergo EpiAcc purely for the research study.
- Patients undergoing EpiAcc for the purposes of a pericardiocentesis.
A Longitudinal Observational Study of Patients Undergoing Therapy for IMISC (TARGET-DERM)
A 5-year Longitudinal Observational Study
• 1. Adults and children (all ages) with Atopic Dermatitis or other Immune-mediated Inflammatory Skin Conditions been prescribed any dermatologic treatment.
• 2. Participant has plans for future visits at the site for continued management of IMISC.
• 1. Inability to provide written informed consent/assent.
• 2. Subjects participating in any interventional study or trial for IMISC treatment trial at the time of enrollment. Patients may be enrolled in TARGET-DERM once participation in the trial is complete. Note: Participants may be enrolled in other registries or studies where IMISC treatment outcomes are observed and/or reported (such as center-based registries).
A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
A Study of the Safety of Unlicensed, Investigational Cord Blood Units Manufactured by the National Cord Blood Program for use in Stem Cell Transplants to Unrelated People
Inclusion Criteria
- Have disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
- Any age and either gender
- Using a cord blood product manufactured by the National Cord Blood Program (at least one, if the graft contains more than one unit)
Exclusion Criteria
- Are only receiving licensed cord blood products
- Are only receiving unlicensed cord blood products from other banks
- Are being transplanted at non-US transplant centers
- Are receiving cord blood products that will be "manipulated" post-thaw (e.g., ex vivo expansion, incubation in vitro, etc.)
Apogee ( addendum to the Medtronic, Inc. Product Surveillance Registry / Destination Therapy (DT) Post Approval Study (PAS) Product Surveillance Registry Platform Addendum
A Study Addendum to the Medtronic, Inc. Product Surveillance Registry/Destination Therapy (DT) Post-Approval Study (PAS) Product Surveillance Registry
- Patient or legally authorized representative provides written authorization and/or consent per institution and geographical requirements.
- Patient has or is intended to receive or be treated with an eligible product.
- Patient is consented within the enrollment window of the treatment/therapy received, refer to the applicable Condition/Therapy Appendix.
- Patient who is, or is expected to be inaccessible for follow-up.
- Patient with exclusion criteria required by local law.
- Patient is currently enrolled in or plans to enroll in any concurrent drug and/or device study that may confound results (i.e., no required intervention that could affect interpretation of all-around product safety and or effectiveness).
Obesity-Related Phenotype Registry : PHENOME REGISTRY
A Study to Establish an Obesity-Related Phenotype Registry
- Age 18 and older.
- Willing to provide consent.
- Men or women.
- Stable weight for 3 months prior to study entry.
- Individuals who are unable to sign consent (e.g., mentally challenged, those declared legally incompetent).
- Recent use of weight loss medications (< 6 months).
- History of abdominal GI surgery other than appendectomy.
- Bleeding disorder.
- Pregnancy.
- Positive history of chronic gastrointestinal diseases, or systemic disease that could affect gastrointestinal motility, or use of medications that may alter gastrointestinal motility, appetite or absorption; e.g., orlistat, phentermine.
- Subject has a known history of any condition or factor judged by the investigator to preclude participation in the study or which might hinder study adherence.
- Any contraindications to MRI.
- Claustrophobia.
Pediatric Obesity Weight Evaluation Registry (POWER) Study (POWER)
• age 18 years or younger
• overweight or obese patient
• initial medical evaluation in a pediatric weight management program between March 1, 2014-April 30, 2020.
• no exclusion criteria
SLIP Registry - SCFE Longitudinal International Prospective Registry (SLIP)
SCFE Longitudinal International Prospective Registry
- Confirmed diagnosis of SCFE.
- Intend to receiving follow-up at participating centre.
- Less than 18 years old at time of admission.
- Adequate diagnostic information and radiographic imaging (for patients previously treated for SCFE).
- No definitive diagnosis of SCFE.
- Do not intend to receive follow-up at a participating centre.
- Greater than 18 years old at time of admission.
- Prior treatment for SCFE not appropriately documented.
- Known or suspected underlying conditions (e.g., cerebral palsy, spina bifida, or osteogenesis imperfecta)
Eligibility last updated 8/18/22. Questions regarding updates should be directed to the study team contact.
Pediatric Spine Registry
A Study to Establish a Pediatric Spine Registry
- Ages 0-40 years old.
- All patients with spine and/or chest wall disorders.
- Age older than 40 years old.
- Patient participates in another registry/database with the specific objective to collect pediatric spine deformity data.
- Family does not want to participate.
Pediatric Spine Registry
A Study to Establish a Pediatric Spine Registry
- Ages 0-40 years old.
- All patients with spine and/or chest wall disorders.
- Age older than 40 years old.
- Patient participates in another registry/database with the specific objective to collect pediatric spine deformity data.
- Family does not want to participate.
Mayo Clinic Radiotherapy Patient Outcomes Registry and Biobanking Study
Mayo Clinic Radiotherapy Patient Outcomes Registry and Biobanking Study
- Treated at Mayo Clinic in Radiation Oncology.
- Radiation therapy for curative intent.
- None.
Mayo Clinic Radiotherapy Patient Outcomes Registry and Biobanking Study
Mayo Clinic Radiotherapy Patient Outcomes Registry and Biobanking Study
- Treated at Mayo Clinic in Radiation Oncology.
- Radiation therapy for curative intent.
- None.
Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry
Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry
- Onset of rheumatic disease prior to age 16 years for JIA and onset prior to age 19 years for all other rheumatic diseases.
- Subject (and/or parent/legal guardian when required) is able to provide written informed consent and willing to comply with study procedures.
- Subject and/or parent/legal guardian can read either English or Spanish.
- Subject and/or parent/legal guardian is willing to be contacted in the future by study staff.
- Greater than 21 years of age at the time of enrollment.
Global Patient Registry of Inherited Retinal Diseases (EYERD Registry)
• Participant has any clinically documented sign(s) and/or symptom(s) consistent with an Inherited Retinal Disease (IRD), or asymptomatic with documented retinal changes detected by imaging or electrophysiology
• Participant has documented genetic variant(s) (known pathogenic, likely pathogenic, or variants of uncertain significance) in relevant genes for any of the following IRDs: X-Linked Retinitis Pigmentosa (XLRP) and/or Achromatopsia (ACHM)
• Participant or legally acceptable representative has provided informed consent (and participant assent, when applicable) in accordance with local requirements
• Participant is able to have relevant visual and/or retinal assessments performed For Caregiver Selection:
• Caregiver has consent from the associated participant to participate in the study, or participant assent and consent from their legally acceptable representative
• Male or female aged greater than or equal to (>=)18 years
• Identified by an enrolled participant (or their legally acceptable representative*) as a primary caregiver
• Caregiver has provided informed consent in accordance with local requirements
•Participant has received a treatment in an IRD-related interventional trial, or is being screened for an IRD-related interventional trial For Caregiver Selection:
•Caregiver has an IRD diagnosis and presents with symptoms (visual impairment)
Transplant Caregiver (CG) Research Registry
Caregiver Registry
- Solid organ transplant candidates (18 years or older) on deferred, active, or inactive transplant waiting lists.
- Nominated adult caregivers (18 years or older) of solid organ transplant patients listed for deceased donor solid organ transplantation.
- CG identified in the electronic medical record.
- Individuals unwilling to provide consent.
- Patients or CG unable to complete questionnaires or consents due to limited English proficiency.
Mayo Clinic Scleroderma Registry
Scleroderma Registry
- Patients with scleroderma meeting either a, b, c, or d:
- the 2013 ACR criteria;
- 3 out of 5 CREST (calcinosis, Raynauds, esophageal dysmotility, sclerodactyly, telangiectasias);
- triad of Raynauds, nailfold capillary abnormalities, and positive scleroderma autoantibody;
- very early diagnosis of systemic sclerosis (VEDOSS) defined as Raynaud’s, puffy digits, and abnormal nailfold capillaries or positive autoantibody.
- Patients with overlapping autoimmune phenotypes may be included, provided they meet the 2013 scleroderma classification criteria (a).
- This protocol will also allow for recruitment of healthy volunteers for comparison studies.
- Patients without scleroderma will be excluded from the primary disease cohort.
- Healthy volunteers(for the comparison cohort) will excluded if they have any autoimmune condition(s) (rheumatic, neurologic, hematologic, etc., at the discretion of the primary investigator or co-investigators).
Eligibility last updated 12/6/21. Questions regarding updates should be directed to the study team contact.
Cervical Spine Research Society (CSRS) Registry Protocols (CSRS)
CSRS Registry
- Consecutive patients age 18 and older with the below diagnoses:
- Cervical radiculopathy or cervical myelopathy;
- Surgical patients eligible for treatment with cervical foraminotomy, cervical arthroplasty, cervical laminectomy, cervical fusion, anterior cervical discectomy and fusion, cervical laminoplasty or a combination of above.
- Individuals < 18 years of age.
- Patients diagnosed at the C1-C2 levels.
Hydronephrosis Registry
A Study to Collect Patient Data Collected Through Standard of Care Clinical Visits on the Subject of Hydronephrosis (Common Pediatric Urological Disorder that is Detected Prenatally, or Postnatal Infancy)
- Patients of age 18 to 65 will be asked to be in this study because they are pregnant and their fetus has hydronephrosis or if they have a child who has hydronephrosis.
- Mothers under 18 will not be enrolled.
- All children will be under the age of 6.
- Anyone who doesn’t carry the hydronephrosis condition.
- Who denies consent.
Undiagnosed Tumor/Undifferentiated Mass Registry (Undiagnosed Tumor/Undifferentiated Mass Registry)
Undiagnosed Tumor/Undifferentiated Mass Registry
- Patient presenting to a General Internal Medicine Service.
- Patient in one of the 3 Mayo Clinic Campuses (Rochester, Arizona, Florida).
- Patient with an undiagnosed mass: New or enlarging lymph nodes clinically or on imaging:
- New mass on imaging of soft tissues, bone, spleen, adrenal gland, retroperitoneum, or intraabdominal location without clear organ association.
- Patient has understood and signed the informed consent to participate in the registry.
- Patient has the ability to complete all aspects of registry enrollment.
- Aged 18 and older.
- Mass involving the breast, brain, kidney, lung, ovary/adnexa, liver, pancreas, sinus, throat, or thyroid gland will be addressed by the respective specialty areas.
- Patients with a known history of any condition or factor judged by the investigator to preclude participation in the registry or which might hinder adherence.
- Lacking the capacity to consent.
- Prisoners or institutionalized individuals.
- Pregnant women.
Eligibility last updated 9/17/21. Questions regarding updates should be directed to the study team contact.
Pulmonary Hypertension Association Registry (PHAR)
A Study to Develop a Pulmonary Hypertension Association Registry (PHAR)
- New patients to a PHA Care Center with either newly diagnosed or established PAH or CTEPH (within 6 months of first outpatient visit at the PHCC).
- Informed consent for data to be abstracted and included in the PHAR provided by the patient or guardian.
- Able to understand and speak either English or Spanish.
- Any exception to the Inclusion Criteria above.
Familial Hypercholesterolemia Identification REgistry (FHIRE) (FHIRE)
A Study to Develop A Familial Hypercholesterolemia Identification REgistry (FHIRE)
- Mayo Clinic patients with suspected Familial Hypercholesterolemia (FH).
- Age ≥ 18 years.
- LDL cholesterol ≥ 190 mg/dl.
- Capable of providing informed consent.
- Lack of research authorization.
- Unable to provide informed consent.
Rochester Older Adult Registry (ROAR) (ROAR)
A Study to Establish a Rochester Older Adult Registry (ROAR)
- Patients had to be seen by a health care provider at least once at the Family Medicine and Primary Care Internal Medicine clinics of Mayo Clinic in Rochester, MN or the Mayo Clinic Health System during the study time.
- Patients had to be empaneled to a primary care provider.
- Ability to give informed consent.
- Ability to read and speak the English language.
- Age 65 or older.
- Patients that had not to be seen by a health care provider during the study time.
- Any participant who refused medical record review.
- Incarcerated individuals.
- Hospice enrollment.
- Memory Care residents.
- Major Neurocognitive disorder (severe and moderate dementia).