- 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
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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Phenylketonuria
- Interventions
- rAvPAL-PEG
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 55 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2019
- U.S. locations
- 14
- States / cities
- Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more
- Conditions
- Mucopolysaccharidosis II, MPS II
- Interventions
- SB-913
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 5
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 2 more
- Conditions
- Gout
- Interventions
- RDEA3170 10 mg, allopurinol 300 mg
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Fabry Disease
- Interventions
- migalastat
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 30 Years to 80 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Gout
- Interventions
- allopurinol, Placebo (sugar pill)
- Drug
- Lead sponsor
- 59th Medical Wing
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- Lackland Air Force Base, Texas
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Timeline
- 2017 – 2027
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- Behavioral Manifestations of Lesch-Nyhan Disease
- Interventions
- sapropterin
- Drug
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Interventions
- AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
- Biological · Procedure · Drug + 3 more
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Gout
- Interventions
- Pegloticase, Methotrexate
- Biological · Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 262 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 74
- States / cities
- Birmingham, Alabama • Anchorage, Alaska • Chandler, Arizona + 63 more
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
- Interventions
- Melphalan
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Gout
- Interventions
- Lesinurad, Placebo, Allopurinol
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 610 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 88
- States / cities
- Mobile, Alabama • Chandler, Arizona • Mesa, Arizona + 76 more
- Conditions
- Colitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 21 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Sjogren-Larsson Syndrome (SLS)
- Interventions
- Not listed
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 2
- States / cities
- Omaha, Nebraska • Pittsburgh, Pennsylvania
- Conditions
- Healthy
- Interventions
- KADIAN Capsule + alcohol (under fasting conditions), KADIAN Capsule + water (under fasting conditions), morphine sulfate IR oral solution + water, KADIAN Capsule + alcohol (under fed conditions)
- Drug
- Lead sponsor
- Actavis Inc.
- Industry
- Eligibility
- 21 Years to 40 Years · Male only
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006
- U.S. locations
- 1
- States / cities
- Lincoln, Nebraska
- Conditions
- Danon Disease
- Interventions
- RP-A501
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
- 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
- Leukodystrophy, Globoid Cell
- Interventions
- PBKR03
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 9 Months
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- MPS IVA, Morquio Syndrome
- Interventions
- Not listed
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 1 Year and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Hyperoxaluria
- Interventions
- Betaine, Placebo
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 6 Years to 70 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Phenylketonurias
- Interventions
- PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
- Device · Diagnostic Test
- Lead sponsor
- Lumos Diagnostics
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Interventions
- sebelipase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2017
- U.S. locations
- 5
- States / cities
- Eureka, California • Sacramento, California • San Francisco, California + 2 more