- Conditions
- Urea Cycle Disorder
- Interventions
- Not listed
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Not listed
- Enrollment
- 203 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2020
- U.S. locations
- 1
- States / cities
- Blue Bell, Pennsylvania
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 73–96
of 1,509
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Gaucher Disease
- Interventions
- OGT 918
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 4 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2007
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Menkes Disease
- Interventions
- Droxidopa Oral Product, Placebo Control
- Drug · Other
- Lead sponsor
- Stephen G. Kaler
- Other
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, Bupropion Hydrochloride
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Cystinosis
- Interventions
- Cysteamine
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 2 Years and older
- Enrollment
- 328 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1986 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Canavan Disease
- Interventions
- AAV9 BBP-812
- Biological
- Lead sponsor
- Aspa Therapeutics
- Industry
- Eligibility
- Up to 30 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 4
- States / cities
- Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
- Conditions
- Parkinson Disease, Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 50 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
- Interventions
- Stem Cell Transplantation
- Procedure
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Blood Coagulation Disorders
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 80 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Pseudohypoparathyroidism Type 1A, Albright Hereditary Osteodystrophy, Pseudopseudohypoparathyroidism
- Interventions
- Neurocognitive and psychosocial testing
- Behavioral
- Lead sponsor
- Connecticut Children's Medical Center
- Other
- Eligibility
- 2 Months to 89 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2030
- U.S. locations
- 1
- States / cities
- Hartford, Connecticut
- Conditions
- Acute Gouty Arthritis
- Interventions
- Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg
- Drug
- Lead sponsor
- Swedish Orphan Biovitrum
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 165 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 37
- States / cities
- Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more
- Conditions
- RCDP - Rhizomelic Chondrodysplasia Punctata, RCDP1, RCDP2, RCDP3, RCDP4, RCDP5
- Interventions
- Not listed
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- Conditions
- Phenylketonuria
- Interventions
- rAvPAL-PEG
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 50 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 8
- States / cities
- Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
- Conditions
- Neuronal Ceroid Lipofuscinosis
- Interventions
- HuCNS-SC
- Biological
- Lead sponsor
- StemCells, Inc.
- Industry
- Eligibility
- 6 Months to 6 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Gaucher Disease
- Interventions
- Digital Engagement Application (GD App), No Intervention
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,257 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2028
- U.S. locations
- 1
- States / cities
- Lexington, Massachusetts
- Conditions
- Gout
- Interventions
- Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1,790 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 192
- States / cities
- Binghamton, Alabama • Birmingham, Alabama • Huntsville, Alabama + 189 more
- 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
- Cerebrotendinous Xanthomatosis (CTX)
- Interventions
- Not listed
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 442 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 44
- States / cities
- Birmingham, Alabama • La Jolla, California • Los Angeles, California + 38 more
- Conditions
- Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
- Interventions
- Screening for FH
- Diagnostic Test
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Phenylketonuria
- Interventions
- BMN 165 (rAvPAL-PEG)
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 70 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Gainesville, Florida • Boston, Massachusetts + 3 more
- Conditions
- Homocystinuria Due to CBS Deficiency
- Interventions
- Not listed
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 1 Year to 65 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 7
- States / cities
- Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
- Conditions
- Cerebrotendinous Xanthomatosis
- Interventions
- Chenodeoxycholic Acid
- Drug
- Lead sponsor
- US Department of Veterans Affairs
- Federal
- Eligibility
- 5 Years to 80 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2009
- U.S. locations
- 1
- States / cities
- East Orange, New Jersey