- 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 217–240
of 501
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Familial Hypercholesterolemia - Heterozygous
- Interventions
- Inclisiran, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 6 Years to 11 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 9
- States / cities
- San Francisco, California • Washington D.C., District of Columbia • Boca Raton, Florida + 4 more
- Conditions
- Lipodystrophy, Familial Partial
- Interventions
- High fat mixed meal
- Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma
- Interventions
- TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning, Dose-Intensified TMZ, Td vaccine, Autologous Hematopoietic Stem Cells (HSC)
- Biological · Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 3 Years to 30 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
- Interventions
- Not listed
- Lead sponsor
- xCures
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Los Altos, California
- Conditions
- Gaucher Disease
- Interventions
- AVR-RD-02
- Drug
- Lead sponsor
- AVROBIO
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Parts A/B: Adrabetadex, Parts A/B: Sham Control
- Drug · Other
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 11 more
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- Egg yolk preparation with cholesterol, Egg substitute, without cholesterol
- Dietary Supplement
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Fabry Disease
- Interventions
- 4D-310
- Biological
- Lead sponsor
- 4D Molecular Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 4
- States / cities
- La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Cascade Screening
- Other
- Lead sponsor
- Northwestern University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Interventions
- Human Placental Derived Stem Cell
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 0 Years to 55 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2022
- U.S. locations
- 3
- States / cities
- Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
- Conditions
- Neimann-Pick Disease
- Interventions
- Vorinostat
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 1
- States / cities
- North Charleston, South Carolina
- 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
- Fed
- Interventions
- CIPRO®XR
- Drug
- Lead sponsor
- Dr. Reddy's Laboratories Limited
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 5,000 participants
- Timeline
- 2016 – 2090
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • Cincinnati, Ohio
- Conditions
- Fabry Disease
- Interventions
- Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
- Conditions
- Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 2,800 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Familial Chylomicronemia Syndrome
- Interventions
- Olezarsen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 1
- States / cities
- Carlsbad, California
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Interventions
- FLT190
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1995 – 2010
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota