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Conditions: Diabetes Mellitus, Type 2, PreDiabetes, Pancreas Cyst, Chronic Pancreatitis, Genetic Predisposition to Disease, Inherited Disease
Interventions: Not listed
Lead sponsor: University of Nebraska; Other
Eligibility: 19 Years and older
Enrollment: 1,250 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2028
U.S. locations: 1
States / cities: Omaha, Nebraska

Conditions: Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Lynch Syndrome, Recurrent Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Primary Central Nervous System Neoplasm, Xeroderma Pigmentosum
Interventions: Biospecimen Collection, Ipilimumab, Nivolumab; Procedure · Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 25 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 7
States / cities: Palo Alto, California • Aurora, Colorado • St Louis, Missouri + 4 more

Conditions: Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
Interventions: Imatinib mesylate; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 148 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2013
U.S. locations: 3
States / cities: Boston, Massachusetts • Portland, Oregon • Philadelphia, Pennsylvania

Conditions: Pelizaeus-Merzbacher Disease
Interventions: ION356; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 17 Years · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 3
States / cities: Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah

Conditions: Phenylketonuria (PKU)
Interventions: JNT-517 Tablet, Placebo; Drug · Other
Lead sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 6
States / cities: Gainesville, Florida • Tampa, Florida • Portland, Oregon + 3 more

Conditions: Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions: diflunisal, placebo; Drug · Other
Lead sponsor: Boston University; Other
Eligibility: 18 Years to 75 Years
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2012
U.S. locations: 3
States / cities: Boston, Massachusetts • Rochester, Minnesota • New York, New York

Conditions: Friedreich Ataxia
Interventions: Omaveloxolone; Drug
Lead sponsor: Biogen; Industry
Eligibility: 16 Years and older · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2035
U.S. locations: 1
States / cities: Morrisville, North Carolina

Conditions: Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions: AAV2CUhCLN2 (3x10^12 particle units); Biological
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 3 Years to 18 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2019
U.S. locations: 1
States / cities: New York, New York

Conditions: Nephrocalcinosis, Renal Calculi, Hypercalciuria, Hyperparathyroidism, Cystinuria
Interventions: videotape for mapping of renal anatomy and papillary biopsy; Other
Lead sponsor: Indiana Kidney Stone Institute; Other
Eligibility: 18 Years and older
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2025
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Prader-Willi Syndrome, Obesity
Interventions: ZGN-440 for Injectable Suspension, ZGN-440 Placebo for Injectable Suspension; Drug
Lead sponsor: Zafgen, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 108 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 15
States / cities: Sacramento, California • San Diego, California • Aurora, Colorado + 12 more

Conditions: Urea Cycle Disorder
Interventions: Acetohydroxamic Acid Oral Tablet, No treatment; Drug · Other
Lead sponsor: Nicholas Ah Mew; Other
Eligibility: 18 Years to 60 Years
Enrollment: 4 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Barth Syndrome
Interventions: Elamipretide, Placebo; Drug
Lead sponsor: Stealth BioTherapeutics Inc.; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Gout, Hyperuricemia
Interventions: Subsidy for food purchases and dietitian education; Other
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years and older
Enrollment: 43 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2019
U.S. locations: 2
States / cities: Woodlawn, Maryland • Boston, Massachusetts

Conditions: Reproductive Effects, Pregnancy
Interventions: Not listed
Lead sponsor: Rowan University; Other
Eligibility: 18 Years and older · Female only
Enrollment: 1,141 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2007
U.S. locations: 1
States / cities: Stratford, New Jersey

Conditions: Phenylketonurias
Interventions: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 14 Years and older
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 14
States / cities: Aurora, Colorado • Gainesville, Florida • Tampa, Florida + 11 more

Conditions: Anemia, Sickle Cell
Interventions: GBT440; Drug
Lead sponsor: Global Blood Therapeutics; Industry
Eligibility: 18 Years to 55 Years · Male only
Enrollment: 7 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 2 Years to 99 Years
Enrollment: 4 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2013
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cascade Testing, Lynch Syndrome, Decision Making, Colorectal Cancer, Uterine Cancer, Cancer Prevention
Interventions: Information Letter and Booklet, Information Letter; Behavioral
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 18 Years and older
Enrollment: 185 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Child, Only, Brain Diseases, Sickle Cell Disease, Anemia, Sickle Cell
Interventions: Carbon Dioxide; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 4 Years to 21 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2026
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Interventions: enzyme analysis; Other
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2012
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Hypophosphatasia
Interventions: Asfotase Alfa; Biological
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 12
States / cities: Hartford, Connecticut • Chicago, Illinois • Boston, Massachusetts + 9 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: ALN-PCSSC, Standard of Care; Drug
Lead sponsor: The Medicines Company; Industry
Eligibility: 12 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Congenital Adrenal Hyperplasia
Interventions: Pre-Screening; Other
Lead sponsor: Adrenas Therapeutics Inc; Industry
Eligibility: 18 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Fabry Disease, Healthy
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Male only
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2000 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

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Blood Markers of Early Pancreas Cancer

Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study

A Study of the Efficacy and Safety of Imatinib Mesylate in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing C-kit Gene

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

The Effect of Diflunisal on Familial Amyloidosis

A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis

Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome

Manipulating the Gut Microbiome Study

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

The Diet Gout Trial

Vitamin D and Pregnancy: Camden Study

A Prospective Clinical Study of Phenylketonuria (PKU)

A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects

The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity

Initial Testing of a Behavioral Intervention About Genetic Services for Families at Risk of Lynch Syndrome

Metabolic and Hemodynamic Reserve in Pediatric SCA

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

A Prospective Sub-Study of the Global Hypophosphatasia Registry

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

PET Scans in Normal Volunteers and Patients With Fabry Disease