- Conditions
- MELAS Syndrome
- Interventions
- Idebenone, Placebo
- Drug · Other
- Lead sponsor
- Michio Hirano
- Other
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
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 1033–1056
of 1,710
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Friedreich Ataxia
- Interventions
- TAK-831, TAK-831 Placebo
- Drug
- Lead sponsor
- Neurocrine Biosciences
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2018
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more
- Conditions
- Metastatic Breast Cancer
- Interventions
- Enobosarm, Exemestane
- Drug
- Lead sponsor
- Veru Inc.
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 34
- States / cities
- Chandler, Arizona • Gilbert, Arizona • Glendale, California + 29 more
- Conditions
- MELAS Syndrome, Mitochondrial Diseases
- Interventions
- Medium-Chain Triglycerides
- Dietary Supplement
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- Not listed
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
- Interventions
- Zavesca (Miglustat)
- Drug
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 6 Months to 5 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Up to 12 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 14
- States / cities
- Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
- Conditions
- CADASIL
- Interventions
- Study Procedures
- Other
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 660 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2027
- U.S. locations
- 12
- States / cities
- Los Angeles, California • San Francisco, California • Denver, Colorado + 9 more
- Conditions
- Epilepsy
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 45 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Canavan Disease
- Interventions
- AAV9 BBP-812
- Biological
- Lead sponsor
- Aspa Therapeutics
- Industry
- Eligibility
- Up to 30 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 4
- States / cities
- Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
- Conditions
- Gaucher Disease
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 14
- States / cities
- La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
- Conditions
- Phenylketonuria
- Interventions
- rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 55 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 11
- States / cities
- Aurora, Colorado • Decatur, Georgia • Chicago, Illinois + 8 more
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
- Interventions
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2029
- U.S. locations
- 1
- States / cities
- Lakewood, California
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin dihydrochloride, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 206 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 26
- States / cities
- La Jolla, California • Los Angeles, California • Palo Alto, California + 23 more
- Conditions
- Wilson's Disease
- Interventions
- VTX-801
- Genetic
- Lead sponsor
- Vivet Therapeutics SAS
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 6
- States / cities
- Sacramento, California • New Haven, Connecticut • Orlando, Florida + 3 more
- Conditions
- Tourette Syndrome, Obsessive-Compulsive Disorder, Sensory Disorders
- Interventions
- None - observational study
- Other
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 250 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
- Interventions
- Not listed
- Lead sponsor
- The Methodist Hospital Research Institute
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Houston, Texas
- 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
- Conditions
- Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
- Interventions
- MBM-01
- Drug
- Lead sponsor
- Matrix Biomed, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Huntington Disease
- Interventions
- Tetrabenazine withdrawal
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Adult Spinal Deformity
- Interventions
- Interventions
- Procedure
- Lead sponsor
- AO Foundation, AO Spine
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 5
- States / cities
- San Francisco, California • Baltimore, Maryland • St Louis, Missouri + 2 more
- Conditions
- Solid Tumors
- Interventions
- thioureidobutyronitrile
- Drug
- Lead sponsor
- Cellceutix Corporation
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Neuroblastoma, Central Nervous System Tumors, Lymphomas, Wilms Tumor
- Interventions
- Stem Cell Transplantation, Busulfan, Melphalan
- Procedure · Drug
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Up to 25 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2009
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
- Interventions
- [18F]florbetaben
- Drug
- Lead sponsor
- Lantheus Germany GmbH
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 244 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 3
- States / cities
- Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania