- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 4 Years to 16 Years · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 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 841–864
of 1,709
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- Conditions
- Amyloid Cardiomyopathy
- Interventions
- Acoramidis
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 9
- States / cities
- San Francisco, California • Stanford, California • New Haven, Connecticut + 6 more
- Conditions
- Spinocerebellar Ataxia Type 3
- Interventions
- Specimen Collection
- Other
- Lead sponsor
- University of Michigan
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 22 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2023
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Interventions
- No interventional drug product utilized in this follow-up study
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 19 Years · Male only
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2038
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 1 more
- Conditions
- Nervous System Malformations, Arthropathy, Neurogenic, Urticaria, Papilledema
- Interventions
- anakinra
- Drug
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Angelman Syndrome
- Interventions
- rugonersen, Sham procedure
- Drug · Procedure
- Lead sponsor
- OHB Pediatrics Ltd.
- Industry
- Eligibility
- 1 Year to 50 Years
- Enrollment
- 165 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 4
- States / cities
- San Diego, California • Miami, Florida • Chicago, Illinois + 1 more
- Conditions
- Fragile X Syndrome, FXS, Fra(X) Syndrome
- Interventions
- BPN14770, Placebo
- Drug
- Lead sponsor
- Tetra Discovery Partners
- Industry
- Eligibility
- 18 Years to 45 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Glycogen Storage Disease, Pompe Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Female only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2024
- U.S. locations
- 2
- States / cities
- Cambridge, Massachusetts • Grand Rapids, Michigan
- Conditions
- Dementia, Dementia, Vascular, Mixed Dementias, Alzheimer Disease, Huntington Disease, Lewy Body Disease, Creutzfeldt-Jakob Syndrome, Frontotemporal Lobar Degeneration, Cognitive Dysfunction, Cognitive Aging, Chronic Disease, Palliative Care, Patient Care, Health Care Quality, Access, and Evaluation, Patient Care, Health Communication, Patient Care Planning, Quality of Life
- Interventions
- Jumpstart Guide
- Behavioral
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,802 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 3
- States / cities
- Seattle, Washington
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
- Interventions
- Neutralizing Antibody to SPK-3006 capsid
- Diagnostic Test
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more
- Conditions
- Fabry Disease
- Interventions
- agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
- Conditions
- Pompe Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Durham, North Carolina • Hershey, Pennsylvania + 3 more
- Conditions
- Ataxia Telangiectasia
- Interventions
- Dexamethasone sodium phosphate
- Drug
- Lead sponsor
- Quince Therapeutics S.p.A.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 101 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Baltimore, Maryland • New Brunswick, New Jersey + 2 more
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
- Interventions
- Cerezyme (imiglucerase for injection)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 10 Years to 65 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2004
- U.S. locations
- 1
- States / cities
- Coral Springs, Florida
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 8 Years to 18 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2010
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 146 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
- Conditions
- Huntington's Disease
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • San Diego, California • Iowa City, Iowa + 5 more
- 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
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
- Interventions
- AB-1003 dose level 1, AB-1003 dose level 2, Placebo
- Genetic · Drug
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 6
- States / cities
- Irvine, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- placebo (saline), Olipudase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2023
- U.S. locations
- 3
- States / cities
- San Francisco, California • Decatur, Georgia • The Bronx, New York
- Conditions
- Growth Hormone Deficiency, Dwarfism
- Interventions
- Dextromethorphan and Caffeine
- Drug
- Lead sponsor
- University of Louisville
- Other
- Eligibility
- 4 Years to 14 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2008
- U.S. locations
- 4
- States / cities
- San Diego, California • Louisville, Kentucky • Kansas City, Missouri + 1 more
- Conditions
- Uveitis, Intermediate Uveitis, Posterior Uveitis, Panuveitis, Noninfectious Uveitis
- Interventions
- triamcinolone acetonide (Triesence®)
- Drug
- Lead sponsor
- Clearside Biomedical, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Omaha, Nebraska • Cleveland, Ohio