- Conditions
- Gaucher Disease
- Interventions
- Blood sample
- Procedure
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 7
- States / cities
- San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 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 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 25–48
of 241
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Gaucher Disease
- Interventions
- eliglustat
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 79 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2017
- U.S. locations
- 3
- States / cities
- Miami, Florida • Saint Paul, Minnesota • Knoxville, Tennessee
- Conditions
- Fabry Disease
- Interventions
- Venglustat (GZ402671), Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 12
- States / cities
- Birmingham, Alabama • Los Angeles, California • Orange, California + 9 more
- Conditions
- Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- LY3884961
- Genetic
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2031
- U.S. locations
- 2
- States / cities
- Durham, North Carolina • Fairfax, Virginia
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Months and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
- Conditions
- Gaucher Disease Type 1
- Interventions
- N-acetylcysteine
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2025
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • New York, New York
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- GZ402665
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- Gaucher Disease
- Interventions
- GA-GCB (velaglucerase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more
- Conditions
- Fabry Disease
- Interventions
- Replagal
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Fairfax, Virginia
- Conditions
- Niemann-Pick Type C Disease
- Interventions
- Nizubaglustat, Placebo
- Drug
- Lead sponsor
- Azafaros B.V.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 72 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 5
- States / cities
- Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
- Conditions
- Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
- Interventions
- 15 O Water
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 123 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1994 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Parkinson Disease
- Interventions
- LY3884961, Methylprednisolone, Sirolimus
- Biological · Drug
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 35 Years to 80 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2031
- U.S. locations
- 11
- States / cities
- Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Interventions
- Not applicable- observational study
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2034
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV®
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 95 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Oakland, California • Atlanta, Georgia + 7 more
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- MGTA-456
- Drug
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Cincinnati, Ohio
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 2
- States / cities
- Rockville, Maryland • Fairfax, Virginia
- Conditions
- Fabry Disease
- Interventions
- PRX-102
- Drug
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 9
- States / cities
- Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase alfa
- Biological
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase-alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
- Conditions
- Fabry Disease
- Interventions
- GZ/SAR402671
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 49 Years · Male only
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia