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Showing 73–96 of 194 matching trials from the live ClinicalTrials.gov search.
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-brve
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
New York, New York
Conditions
Spinal Muscular Atrophy
Interventions
Valproic Acid (VPA), Placebo
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 60 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Incomplete Spinal Cord Injury
Interventions
EA-KAFO
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
3 Years to 17 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Completed Not applicable Interventional Results available

Progressive Strength Training in Spinal Muscular Atrophy

NCT01233817
Conditions
Muscular Atrophy, Spinal
Interventions
Progressive strength training
Other
Lead sponsor
University of Utah
Other
Eligibility
5 Years to 21 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Conditions
Spinal Muscular Atrophy 1
Interventions
AVXS-101
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Recruiting No phase listed Observational

Gait and Bone Health in SMA

NCT07400198
Conditions
Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Jacqueline Montes
Other
Eligibility
8 Years to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New York, New York
Conditions
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophies
Interventions
BrainGate Neural Interface System
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Sacramento, California
Conditions
Muscle Weakness, Problems Moving Their Arms and Legs
Interventions
Extension assist knee ankle foot orthosis (EA-KAFO)
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
5 Years to 25 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years and older
Enrollment
238 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
25
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more
Conditions
Muscular Atrophy, Spinal
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 54 Years · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
4
States / cities
Palo Alto, California • Boston, Massachusetts • Cambridge, Massachusetts + 1 more
Completed No phase listed Observational Accepts healthy volunteers

A Pilot Study of Biomarkers for Spinal Muscular Atrophy

NCT00756821
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
2 Years to 12 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
16
States / cities
Birmingham, Alabama • Stanford, California • Aurora, Colorado + 13 more
Conditions
Muscular Atrophy, Spinal
Interventions
Salanersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
0 Days to 42 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
3
States / cities
Chicago, Illinois • Flower Mound, Texas • Norfolk, Virginia
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
6 Months to 60 Months
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Stanford, California • Orlando, Florida + 8 more
Conditions
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 99 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Castration-resistant Prostate Cancer, Metastatic Cancer, Prostate Cancer
Interventions
pTVG-HP, pTVG-AR, Pembrolizumab
Biological · Drug
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
St Louis, Missouri • Madison, Wisconsin
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
Vanderbilt University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Nashville, Tennessee
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
Physical Disability
Interventions
EnhanceWellness for Disability (EW-D), Wellness Education
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
45 Years to 64 Years
Enrollment
516 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Adult Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Great Neck, New York
Conditions
Spinal and Bulbar Muscular Atrophy
Interventions
BVS857, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Orange, California • Bethesda, Maryland • Columbus, Ohio
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Completed Not applicable Interventional Results available

Glutathione and Health With Post-Polio Syndrome

NCT01402570
Conditions
Post-polio Syndrome, Physical Activity, Depression, Pain, Sleep Disorders
Interventions
Glutathione
Dietary Supplement
Lead sponsor
University of Michigan
Other
Eligibility
50 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Ann Arbor, Michigan