- Conditions
- Charcot Marie Tooth Disease
- Interventions
- Placebo, Mexiletine
- Other · Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Rochester, New York
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- University of Kansas Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 2
- States / cities
- Kansas City, Kansas • Salt Lake City, Utah
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Interventions
- L-arginine
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 7 Years to 11 Years · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
- Interventions
- Abilitech Assist
- Device
- Lead sponsor
- AbiliTech Medical Inc.
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Saint Paul, Minnesota
- Conditions
- Cystic Fibrosis
- Interventions
- Sildenafil (Acute-1 hour), Sildenafil (Subchronic-4 weeks), Placebo
- Drug
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 100 Years
- Enrollment
- 999 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fibromyalgia, Irritable Bowel Syndrome, Chronic Fatigue Syndrome, Depression
- Interventions
- Not listed
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 560 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 4
- States / cities
- Peoria, Illinois • Cincinnati, Ohio • Cleveland, Ohio + 1 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Pamrevlumab, Placebo, Corticosteroids
- Drug
- Lead sponsor
- Kyntra Bio
- Industry
- Eligibility
- 6 Years to 11 Years · Male only
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 27
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 24 more
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 15 Years · Male only
- Enrollment
- 219 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 21
- States / cities
- Los Angeles, California • Sacramento, California • Stanford, California + 18 more
- Conditions
- Limb-Girdle Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 8
- States / cities
- Atlanta, Georgia • Iowa City, Iowa • Kansas City, Kansas + 5 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
- ALS
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 254 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 13
- States / cities
- Los Angeles, California • Aurora, Colorado • Ann Arbor, Michigan + 10 more
- Conditions
- Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis, Polio and Post-polio Syndrome, Syringomyelia, Spinal Stenosis, Spinal Neoplasms, Spinal Cord Diseases
- Interventions
- SCI Hard
- Behavioral
- Lead sponsor
- University of Michigan
- Other
- Eligibility
- 13 Years to 29 Years
- Enrollment
- 184 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- WVE-210201, Placebo
- Drug
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Industry
- Eligibility
- 5 Years to 18 Years · Male only
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- MNK-1411, Placebo
- Drug · Other
- Lead sponsor
- Mallinckrodt ARD LLC
- Industry
- Eligibility
- 4 Years to 8 Years · Male only
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 5
- States / cities
- Gulf Breeze, Florida • Atlanta, Georgia • Nashville, Tennessee + 2 more
- Conditions
- Muscular Dystrophies, Limb-Girdle
- Interventions
- SRP-9005, Corticosteroid
- Biological · Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Norfolk, Virginia
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, PLACEBO
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 360 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
- Interventions
- Alglucosidase Alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 9
- States / cities
- Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more
- Conditions
- Prostate Cancer
- Interventions
- Standard of care ADT + (HMB + arginine + glutamine), Standard of care ADT
- Drug · Other
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 60 Years and older · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2021
- U.S. locations
- 1
- States / cities
- Milwaukee, Wisconsin
- Conditions
- Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Dystrophia Myotonica, Dystrophia Myotonica 1, Dystrophia Myotonica 2, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Myotonic Myopathy, Proximal, PROMM (Proximal Myotonic Myopathy), Proximal Myotonic Myopathy, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonia Atrophica
- Interventions
- Patient-entered data
- Other
- Lead sponsor
- Myotonic Dystrophy Foundation
- Other
- Eligibility
- Not listed
- Enrollment
- 3,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- Westat
- Other
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008
- U.S. locations
- 5
- States / cities
- Stanford, California • St Louis, Missouri • New York, New York + 2 more
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier, Dystrophinopathy Female Carrier
- Interventions
- Not listed
- Lead sponsor
- The Duchenne Registry
- Other
- Eligibility
- Not listed
- Enrollment
- 10,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2047
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older
- U.S. locations
- 42
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 36 more
- Conditions
- Spinal Muscular Atrophy
- Interventions
- OAV101
- Genetic
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • St Louis, Missouri