- Conditions
- Mucolipidosis Type IV
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 1 Year to 64 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2021
- U.S. locations
- 1
- States / cities
- Dallas, Texas
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 1441–1464
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Bile Acid Synthesis Disorders
- Interventions
- Cholbam
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2039
- U.S. locations
- 24
- States / cities
- Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl 20 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 6
- States / cities
- Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more
- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Acute Gout
- Interventions
- apremilast, indomethacin SR
- Drug
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Other
- Eligibility
- 18 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 1
- States / cities
- Lebanon, New Hampshire
- 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
- Amyloid Cardiomyopathy, Transthyretin-Related
- Interventions
- Acoramidis (AG10)
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 389 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 26
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
- Conditions
- Mucopolysaccharidoses
- Interventions
- Not listed
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 5 Years to 35 Years
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 3
- States / cities
- Oakland, California • Torrance, California • Minneapolis, Minnesota
- Conditions
- X-Linked Hypophosphatemia
- Interventions
- KRN23
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- BMN 307
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Morristown, New Jersey
- Conditions
- Progeria, Hutchinson-Gilford Syndrome
- Interventions
- Lonafarnib
- Drug
- Lead sponsor
- Monica E. Kleinman
- Other
- Eligibility
- 1 Year and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Classic Galactosemia
- Interventions
- AT-007, Placebo
- Drug
- Lead sponsor
- Applied Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 114 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2021
- U.S. locations
- 4
- States / cities
- Anaheim, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
- Conditions
- Hematologic Malignancies
- Interventions
- UCB and HPDSC
- Procedure
- Lead sponsor
- Celgene Corporation
- Industry
- Eligibility
- Up to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana
- Conditions
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
- Interventions
- Growth Hormone, Whole body Protein turnover, DEXA scan
- Drug · Procedure
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 3 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Autism, Asperger Disorder, PDD-NOS
- Interventions
- Cholesterol
- Other
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Phenylketonuria
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 111 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Sacramento, California • San Jose, California + 11 more
- Conditions
- Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
- Interventions
- Theophylline ER
- Drug
- Lead sponsor
- Jaclyn Tamaroff
- Other
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2026
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Cerliponase Alfa
- Drug
- Lead sponsor
- David L Rogers, MD
- Other
- Eligibility
- 24 Months to 72 Months
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Glycogen Storage Disease Type IA
- Interventions
- DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 9
- States / cities
- Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years to 60 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1995
- U.S. locations
- 5
- States / cities
- San Diego, California • San Francisco, California • New York, New York
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Tildacerfont/Placebo
- Drug
- Lead sponsor
- Spruce Biosciences
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Diego, California + 13 more
- Conditions
- Arthritis, Rheumatoid Arthritis (RA), Gout, Fibromyalgia (FM), Osteoarthritis, Lupus Erythematosus, Systemic
- Interventions
- DREAM Adaptive Intervention
- Behavioral
- Lead sponsor
- University of South Carolina
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 285 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Columbia, South Carolina
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Zidovudine, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1990
- U.S. locations
- 3
- States / cities
- San Diego, California • New York, New York • Rochester, New York
- Conditions
- Lactose Intolerance
- Interventions
- RP-G28 or placebo
- Drug
- Lead sponsor
- Ritter Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011
- U.S. locations
- 2
- States / cities
- Honolulu, Hawaii • Dallas, Texas