- Conditions
- Parkinson Disease, Gaucher Disease
- Interventions
- 15-0 H20
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years to 110 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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 793–816
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2009
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Interventions
- ALLN-346, Placebo
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 17
- States / cities
- Birmingham, Alabama • Muscle Shoals, Alabama • Sheffield, Alabama + 12 more
- Conditions
- Gout
- Interventions
- Denosumab
- Drug
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 30 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 1
- States / cities
- Birmingham, Alabama
- Conditions
- Gaucher Disease
- Interventions
- AVR-RD-02
- Biological
- Lead sponsor
- John Bernat
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2037
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Pompe Disease
- Interventions
- respiratory muscle strength training
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 3 Years to 99 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2012
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- virtual educational based transition intervention self-paced
- Other
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 16 Years to 22 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
- Interventions
- Carbaglu, Placebo
- Drug
- Lead sponsor
- Mendel Tuchman
- Other
- Eligibility
- 1 Week to 99 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2020
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase-alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
- Conditions
- Generalized Lipodystrophy
- Interventions
- Mibavademab, Placebo
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 2
- States / cities
- Ann Arbor, Michigan • Dallas, Texas
- Conditions
- Phenylketonuria (PKU)
- Interventions
- MZE782, Placebo
- Drug · Other
- Lead sponsor
- Maze Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 7
- States / cities
- Oakland, California • Palo Alto, California • Chicago, Illinois + 4 more
- Conditions
- Ornithine Transcarbamylase (OTC) Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Timeline
- 2018 – 2029
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Pseudotumor Cerebri, Cystinosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Congenital Adrenal Hyperplasia, 21-OHD
- Interventions
- Tildacerfont
- Drug
- Lead sponsor
- Spruce Biosciences
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 13
- States / cities
- Sacramento, California • San Diego, California • Chicago, Illinois + 10 more
- Conditions
- Gout
- Interventions
- Storytelling
- Behavioral
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 4
- States / cities
- Birmingham, Alabama • Iowa City, Iowa • Boston, Massachusetts + 1 more
- Conditions
- Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
- Interventions
- atumelnant (CRN04894)
- Drug
- Lead sponsor
- Crinetics Pharmaceuticals Inc.
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 3
- States / cities
- Minneapolis, Minnesota • Morehead City, North Carolina • Philadelphia, Pennsylvania
- Conditions
- Achondroplasia
- Interventions
- Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 11 Years
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 5
- States / cities
- Oakland, California • Wilmington, Delaware • Baltimore, Maryland + 2 more
- 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
- Familial Partial Lipodystrophy, Nonalcoholic Steatohepatitis, NAFLD
- Interventions
- Metreleptin
- Drug
- Lead sponsor
- University of Michigan
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Pompe Disease
- Interventions
- Diaphragm conditioning via phrenic nerve stimulation
- Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 2 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- MPS VI
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Hypercholesterolemia, Familial
- Interventions
- ISIS 301012
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 5
- States / cities
- Chicago, Illinois • New York, New York • Winston-Salem, North Carolina + 2 more
- Conditions
- Idiopathic Pulmonary Fibrosis
- Interventions
- No intervention
- Other
- Lead sponsor
- 23andMe, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 690 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Sunnyvale, California
- Conditions
- Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia
- Interventions
- VERVE-201
- Drug
- Lead sponsor
- Verve Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 4
- States / cities
- Clearwater, Florida • Miami Lakes, Florida • Port Orange, Florida + 1 more