- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- diet
- Behavioral
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Portland, Oregon
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 193–216
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Phenylketonuria, Behavior and Behavior Mechanisms, PAH Gene Expression
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- Tulane University School of Medicine
- Other
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana
- Conditions
- Gout
- Interventions
- Tart cherry extract 60 mL, Tart cherry extract 120 mL
- Dietary Supplement
- Lead sponsor
- Rutgers, The State University of New Jersey
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- New Brunswick, New Jersey
- Conditions
- Lesch-Nyhan Disease
- Interventions
- Ecopipam
- Drug
- Lead sponsor
- Psyadon Pharma
- Industry
- Eligibility
- 6 Years to 65 Years · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 2
- States / cities
- San Diego, California • Atlanta, Georgia
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm, Mucopolysaccharidosis I
- Interventions
- Venipuncture
- Procedure
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Cardiac Amyloidosis
- Interventions
- AI ECG Amyloid algorithm
- Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20,143 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 4
- States / cities
- Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 1 more
- Conditions
- Mucopolysaccharidosis VI
- Interventions
- N-acetylgalactosamine 4-sulfatase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2005
- U.S. locations
- 1
- States / cities
- Novato, California
- 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
- X-linked Hypophosphatemia
- Interventions
- KRN23
- Drug
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, agalsidase
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
- Conditions
- Glycogen Storage Disease Type III
- Interventions
- UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker
- Biological · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 4
- States / cities
- Orange, California • Atlanta, Georgia • Philadelphia, Pennsylvania + 1 more
- Conditions
- Fabry Disease
- Interventions
- agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
- 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
- Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS), Cancer
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 80 Years
- Enrollment
- 88 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2021
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • Decatur, Georgia • Detroit, Michigan
- Conditions
- Congenital Adrenal Hyperplasia, Healthy
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 9 Years to 40 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypophosphatasia
- Interventions
- asfotase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 13 Years to 65 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2016
- U.S. locations
- 2
- States / cities
- St Louis, Missouri • Durham, North Carolina
- 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
- Zellweger Spectrum
- Interventions
- Not listed
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 92 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Methylmalonic Acidemia
- Interventions
- mRNA-3705, Placebo
- Biological
- Lead sponsor
- ModernaTX, Inc.
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Palo Alto, California • Philadelphia, Pennsylvania
- Conditions
- Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
- Interventions
- Not listed
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 1 Day to 120 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2054
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland
- Conditions
- Wilson Disease
- Interventions
- Standard of Care Medications
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
- Conditions
- Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 1
- States / cities
- Durham, North Carolina