- Conditions
- Type 1 Gaucher Disease
- Interventions
- AT2101
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- Miramar, Florida
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 481–504
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Argininosuccinic Aciduria, Argininosuccinate Lyase Deficiency, Urea Cycle Disorders
- Interventions
- Nitric oxide supplement, Placebo
- Dietary Supplement
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 8 Years to 64 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Gout
- Interventions
- Lesinurad, Placebo
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 20 Years to 55 Years · Male only
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Glendale, California
- Conditions
- Iron Overload, Excessive Body Iron Burden
- Interventions
- R2*-UTE, R2*-GRE, Liver biopsy
- Device · Procedure
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 142 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Gout
- Interventions
- RDEA3170 10 mg, RDEA3170 2.5 mg
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Gaucher Disease Type 1
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 16 Years to 90 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- Phenylketonuria
- Interventions
- Kuvan, Meal Challenge
- Drug · Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 10 Years to 45 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Pompe Disease
- Interventions
- BMN 701
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 4
- States / cities
- San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more
- Conditions
- Lipoidosis
- Interventions
- SCH-58235
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Apparent Mineralocorticoid Excess (AME)
- Interventions
- Glycyrrhetic Acid, Placebo
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2002 – 2009
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Pelizaeus-Merzbacher Disease
- Interventions
- ION356
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 17 Years · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah
- Conditions
- Healthy
- Interventions
- KADIAN (morphine sulfate sustained-release) capsules, Avinza (morphine sulfate sustained-release) capsules
- Drug
- Lead sponsor
- Actavis Inc.
- Industry
- Eligibility
- 19 Years to 40 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004
- U.S. locations
- 1
- States / cities
- Lincoln, Nebraska
- Conditions
- Gaucher Disease, Type 1
- Interventions
- velaglucerase alfa
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years and older
- U.S. locations
- 32
- States / cities
- Phoenix, Arizona • Beverly Hills, California • La Jolla, California + 28 more
- Conditions
- Gout, Hyperuricemia
- Interventions
- Subsidy for food purchases and dietitian education
- Other
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 43 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 2
- States / cities
- Woodlawn, Maryland • Boston, Massachusetts
- Conditions
- Wilson Disease
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
- Conditions
- Fabry Disease
- Interventions
- Fluorescein angiography
- Other
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Methylmalonic Acidemia
- Interventions
- mRNA-3705
- Drug
- Lead sponsor
- ModernaTX, Inc.
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2034
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Palo Alto, California • San Diego, California
- Conditions
- Phenylketonurias
- Interventions
- sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 89 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 13
- States / cities
- Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
- Conditions
- Leigh Syndrome
- Interventions
- Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Sanfilippo Syndrome
- Interventions
- Recombinant human heparan N-sulfatase [rhHNS]
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 12 Months to 48 Months
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 3
- States / cities
- Torrance, California • Minneapolis, Minnesota • Chapel Hill, North Carolina
- Conditions
- X-linked Hypophosphatemia
- Interventions
- KK8123
- Drug
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 3
- States / cities
- Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
- Interventions
- Not listed
- Lead sponsor
- Fairview University Medical Center
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota