- Conditions
- Gaucher's Disease
- Interventions
- glucocerebrosidase
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Pittsburgh, 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 769–792
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
- Interventions
- Hematopoietic stem cell transplantation
- Procedure
- Lead sponsor
- Children's Hospital Los Angeles
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Hypophosphatasia
- Interventions
- ALXN1850
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 4
- States / cities
- Las Vegas, Nevada • Columbus, Ohio • Nashville, Tennessee + 1 more
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Jerry Vockley, MD, PhD
- Other
- Eligibility
- 1 Month and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Enriched Hematopoetic Stem Cell Infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- 3 Years and older · Male only
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Cardiac Amyloidosis
- Interventions
- 18F-Florbetaben (Neuraceq®) PET/MRI
- Drug
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Fabry Disease
- Interventions
- Functional MRI and fast bound-pool fraction imaging
- Other
- Lead sponsor
- University of Utah
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Wilson Disease
- Interventions
- ALXN1840, Celecoxib
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Cardiac Amyloidosis, Pacemaker
- Interventions
- Pacemaker Rate
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Akron, Ohio
- Conditions
- Gout, Hyperuricemia
- Interventions
- Uricase-PEG 20
- Biological
- Lead sponsor
- EnzymeRx
- Industry
- Eligibility
- 40 Years to 75 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2010
- U.S. locations
- 2
- States / cities
- Miami, Florida • Duncansville, Pennsylvania
- Conditions
- Gastrointestinal, Colic
- Interventions
- Lactobacillus reuteri, Placebo
- Biological
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- 3 Weeks to 3 Months
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Houston, Texas
- 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
- Hypertriglyceridemia, Gout, Pericarditis
- Interventions
- Colchicine
- Drug
- Lead sponsor
- Scripps Translational Science Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Fructose Intolerance
- Interventions
- Placebo, Alanine
- Dietary Supplement
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2026
- U.S. locations
- 16
- States / cities
- San Diego, California • Stanford, California • Aurora, Colorado + 12 more
- Conditions
- Amyloidosis, Paraproteinemias, Cardiomyopathies
- Interventions
- Amyloidosis, Cardiac Amyloidosis
- Other
- Lead sponsor
- University of Cincinnati
- Other
- Eligibility
- 18 Years to 85 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Gout, Chronic Kidney Diseases
- Interventions
- allopurinol capsule, 100-800 mg by mouth once daily, febuxostat tablet 40-120 mg by mouth once daily, Placebo, vehicle control (febuxostat-shaped), Placebo, vehicle control (allopurinol-shaped)
- Drug
- Lead sponsor
- VA Office of Research and Development
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 950 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 23
- States / cities
- Loma Linda, California • San Diego, California • San Francisco, California + 19 more
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
- Interventions
- AG10, Placebo
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 90 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- X-linked Hypophosphatemia
- Interventions
- burosumab
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- 5 Years to 12 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 4
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 1 more
- Conditions
- Fabry Disease
- Interventions
- Lucerastat
- Drug
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 107 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2029
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • Irvine, California • Oakland, California + 10 more
- Conditions
- Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis, Type2 Diabetes, Familial Hypercholesterolemia
- Interventions
- Inclisiran, Evolocumab
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 382 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 8
- States / cities
- Jacksonville, Florida • Indianapolis, Indiana • New York, New York + 4 more
- Conditions
- Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy, Insulin Resistance
- Interventions
- Leptin, Placebo
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 6 Years to 70 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, NaPBA
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more