- Conditions
- Mitochondrial Diseases
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 16
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 12 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 200
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Inherited Mitochondrial Disease
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 101 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 14
- States / cities
- La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Female only
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Leukemia, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Psychosocial Effects of Cancer and Its Treatment, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Interventions
- psychosocial assessment and care
- Procedure
- Lead sponsor
- Children's Oncology Group
- Network
- Eligibility
- Up to 21 Years
- Enrollment
- 359 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2005
- U.S. locations
- 26
- States / cities
- Sacramento, California • Gainesville, Florida • Miami, Florida + 23 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
- 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
- ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 338 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Conditions
- Granulomatosis With Polyangiitis, Microscopic Polyangiitis, ANCA Associated Vasculitis
- Interventions
- Obinutuzumab, Rituximab
- Drug
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Rochester, Minnesota • Philadelphia, Pennsylvania
- 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
- ANCA Associated Vasculitis (AAV)
- Interventions
- Rapcabtagene autoleucel, Active Comparator, Glucocorticoids
- Biological · Other · Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 14
- States / cities
- Aurora, Colorado • Jacksonville, Florida • Chicago, Illinois + 10 more
- Conditions
- Mitochondrial Disease
- Interventions
- N-Acetylcysteine
- Drug
- Lead sponsor
- Michio Hirano, MD
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Stroke, Carotid Stenosis
- Interventions
- carotid endarterectomy
- Procedure
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 6,900 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Interventions
- agalsidase beta
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2024
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
- Conditions
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Microscopic Polyangiitis, Wegener Granulomatosis
- Interventions
- Rituximab, Azathioprine
- Biological · Drug
- Lead sponsor
- Cambridge University Hospitals NHS Foundation Trust
- Other
- Eligibility
- 15 Years and older
- Enrollment
- 188 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Ann Arbor, Michigan • Rochester, Minnesota + 6 more
- Conditions
- Hand Function, Stroke, Upper Extremity, Hemiparesis, Cerebrovascular Accident, Stroke Rehabilitation
- Interventions
- Real Stimulation, Sham Stimulation
- Device
- Lead sponsor
- TheraBracelet, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Charleston, South Carolina
- Conditions
- Fabry Disease, Proteinuria
- Interventions
- enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers
- Drug
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 19 Years to 85 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 2 more
- Conditions
- MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 4 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl, ERT
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Atlanta, Georgia + 5 more