- Conditions
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
- Interventions
- Not listed
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 7 Years to 18 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1273–1296
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
- Interventions
- no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Not listed
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 3
- States / cities
- Orange, California • Ann Arbor, Michigan • Durham, North Carolina
- Conditions
- Gaucher Disease Type 1, Gaucher Disease Type 3
- Interventions
- Velaglucerase alfa
- Drug
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 4 Years to 14 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Interventions
- Allo-HSCT
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
- Conditions
- Phenylketonuria
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Wilson Disease
- Interventions
- UX701, Standard of Care (SOC)
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 82 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2034
- U.S. locations
- 12
- States / cities
- Los Angeles, California • Redwood City, California • Sacramento, California + 9 more
- Conditions
- Phenylketonuria
- Interventions
- Administration of CANTAB and Subject Global Assessment
- Other
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Lexington, Kentucky • Omaha, Nebraska • Oklahoma City, Oklahoma + 1 more
- Conditions
- Propionic Acidemia
- Interventions
- ornithine alpha ketoglutarate, glutamine, disodium citrate
- Drug
- Lead sponsor
- Nicola Longo
- Other
- Eligibility
- 5 Years to 12 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Pancreatic Cancer
- Interventions
- Gemcitabine, TT vaccine booster
- Drug · Biological
- Lead sponsor
- Albert Einstein College of Medicine
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- The Bronx, New York
- Conditions
- Chronic Gout Refractory to Conventional Therapy
- Interventions
- pegloticase 8 mg i.v.
- Drug
- Lead sponsor
- Savient Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • Wheaton, Maryland • Durham, North Carolina + 1 more
- Conditions
- Wilson Disease
- Interventions
- ALXN1840
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 4
- States / cities
- Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 1 more
- Conditions
- Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Cerliponase Alfa, Administration Kit
- Drug · Device
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 16
- States / cities
- Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
- Conditions
- Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Statin Adverse Reaction
- Interventions
- bempedoic acid, placebo
- Drug · Other
- Lead sponsor
- Esperion Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 345 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 52
- States / cities
- Gilbert, Arizona • Anaheim, California • Long Beach, California + 47 more
- Conditions
- Fabry Disease
- Interventions
- Agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 5 Years to 18 Years · Male only
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Cincinnati, Ohio • Seattle, Washington
- Conditions
- Cerebrovascular Accident, Fabry Disease, Healthy
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1997 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Evolocumab
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
- Interventions
- Non-interventional.
- Other
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 1
- States / cities
- North Charleston, South Carolina
- Conditions
- Pompe Disease
- Interventions
- Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
- Biological · Drug · Other
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 15
- States / cities
- Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- Conditions
- Familial Hypercholesterolemia, Adherence, Medication
- Interventions
- Monetary incentives
- Behavioral
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 12 Years to 21 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Kidney Calculi
- Interventions
- Potassium Citrate, Acetazolamide, Diet low in oxalate and purines
- Drug · Behavioral
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Other
- Eligibility
- 5 Years to 17 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2007
- U.S. locations
- 1
- States / cities
- Kansas City, Missouri
- Conditions
- Maculopathy, Age-Related, Choroidal Neovascularization
- Interventions
- Not listed
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 4
- States / cities
- Beverly Hills, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
- Conditions
- Gout
- Interventions
- Bucillamine, Colchicine
- Drug
- Lead sponsor
- Revive Therapeutics, Ltd.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015
- U.S. locations
- 3
- States / cities
- San Roman, California • Arlington, Texas • San Antonio, Texas