- Conditions
- Limb-Girdle Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 277 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2018
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
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 1–24
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Charcot Marie Tooth Disease
- Interventions
- Not listed
- Lead sponsor
- University of Iowa
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2025
- U.S. locations
- 9
- States / cities
- Palo Alto, California • Hartford, Connecticut • Orlando, Florida + 6 more
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Interventions
- NeuRx® Diaphragm Pacing System™ (DPS)
- Device
- Lead sponsor
- Barrow Neurological Institute
- Other
- Eligibility
- 21 Years and older
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 25
- States / cities
- Phoenix, Arizona • Los Angeles, California • San Diego, California + 21 more
- Conditions
- Muscular Dystrophies
- Interventions
- rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
- Genetic
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Amyotrophic Lateral Sclerosis, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Interventions
- Not listed
- Lead sponsor
- University of Miami
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2034
- U.S. locations
- 11
- States / cities
- Irvine, California • Palo Alto, California • San Francisco, California + 8 more
- Conditions
- Myasthenia Gravis
- Interventions
- Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)
- Drug
- Lead sponsor
- Muscular Dystrophy Association
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Spironolactone, Prednisolone
- Drug
- Lead sponsor
- Kevin Flanigan
- Other
- Eligibility
- 4 Years to 7 Years · Male only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 4
- States / cities
- Iowa City, Iowa • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Interventions
- FSHD-specific functional rating scale, Electrical Impedance Myography
- Diagnostic Test · Device
- Lead sponsor
- University of Kansas Medical Center
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 324 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Kansas City, Kansas • Baltimore, Maryland + 5 more
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- lithium carbonate
- Drug
- Lead sponsor
- Forbes Norris MDA/ALS Research Center
- Other
- Eligibility
- 21 Years to 80 Years
- Enrollment
- 109 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 10
- States / cities
- Scottsdale, Arizona • Los Angeles, California • Orange, California + 7 more
- Conditions
- Familial Amyotrophic Lateral Sclerosis
- Interventions
- Pyrimethamine
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 2
- States / cities
- New York, New York • Houston, Texas
- Conditions
- Charcot-Marie-Tooth Disease, Type Ia
- Interventions
- Ascorbic acid (Vitamin C), placebo
- Drug
- Lead sponsor
- Wayne State University
- Other
- Eligibility
- 13 Years to 70 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 3
- States / cities
- Baltimore, Maryland • Detroit, Michigan • Rochester, New York
- Conditions
- Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 8 Years to 55 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2021
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- ALS Registry
- Behavioral
- Lead sponsor
- Forbes Norris MDA/ALS Research Center
- Other
- Eligibility
- Not listed
- Enrollment
- 15,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- sodium phenylbutyrate
- Drug
- Lead sponsor
- US Department of Veterans Affairs
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 10
- States / cities
- Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 7 more
- Conditions
- Mitochondrial DNA Depletion Syndrome 2 Myopathic Type, Thymidine Kinase 2 Deficiency
- Interventions
- Thymidine
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
- Interventions
- Screening
- Other
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 448 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2015
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 6 Years to 18 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 3
- States / cities
- Washington D.C., District of Columbia • St Louis, Missouri • Charlotte, North Carolina
- Conditions
- Becker Muscular Dystrophy
- Interventions
- Tadalafil, Placebo
- Drug
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2012
- U.S. locations
- 2
- States / cities
- Los Angeles, California
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- PTC124
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 3
- States / cities
- Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
- Conditions
- TRPV4 Gene Mutation
- Interventions
- Not listed
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 3 Years to 80 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2048
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- rhIGF-I/rhIGFBP-3, placebo
- Drug
- Lead sponsor
- Insmed Incorporated
- Industry
- Eligibility
- 21 Years to 65 Years
- Enrollment
- 69 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 12
- States / cities
- Orange, California • Sacramento, California • Kansas City, Kansas + 9 more
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Oxepa, Jevity 1.5, Jevity 1.0
- Dietary Supplement
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Irvine, California • San Francisco, California + 10 more
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Creatine
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years to 80 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- St Louis, Missouri