- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Interventions
- Levetiracetam
- Biological
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
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 97–120
of 164
matching trials from the live ClinicalTrials.gov search.
- 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
- Spinal Muscular Atrophy
- Interventions
- Not listed
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 17 Years to 70 Years
- Enrollment
- 67 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- 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
- 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
- Muscular Atrophy, Spinal
- Interventions
- Nusinersen
- Drug
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 7 Days and older
- Enrollment
- 145 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 7
- States / cities
- Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 4 more
- Conditions
- Prostate Cancer, Prostatic Neoplasms
- Interventions
- Panobinostat, Bicalutamide
- Drug
- Lead sponsor
- NYU Langone Health
- Other
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 4
- States / cities
- New Brunswick, New Jersey • Lake Success, New York • New York, New York + 1 more
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Not listed
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- Not listed
- Enrollment
- 4,114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- East Hanover, New Jersey
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Salanersen, Sham Procedure
- Drug · Procedure
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 0 Days to 7 Months
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2033
- U.S. locations
- 1
- States / cities
- Flower Mound, Texas
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- Not listed
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2037
- U.S. locations
- 1
- States / cities
- Columbia, Maryland
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- ARGX-119 IV, Placebo IV
- Biological · Other
- Lead sponsor
- argenx
- Industry
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 16
- States / cities
- Little Rock, Arkansas • San Diego, California • Stanford, California + 13 more
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4, SMA
- Interventions
- Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads)
- Device
- Lead sponsor
- Marco Capogrosso
- Other
- Eligibility
- 16 Years to 65 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2029
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Spinal Muscular Atrophy
- Interventions
- NMD670, Placebo
- Drug
- Lead sponsor
- NMD Pharma A/S
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Palo Alto, California • Kansas City, Kansas + 6 more
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Hydroxyurea, Placebo to match hydroxyurea
- Drug
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 1 Year to 10 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2010
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Risdiplam
- Drug
- Lead sponsor
- Bakri Elsheikh
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Spinal Muscular Atrophy 1
- Interventions
- Onasemnogene Abeparvovec-xioi
- Biological
- Lead sponsor
- Novartis Gene Therapies
- Industry
- Eligibility
- Not listed
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2030
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
- Interventions
- Not listed
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 17,667 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
- Interventions
- Observational
- Other
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2023
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- Conditions
- Spinal Muscular Atrophy
- Interventions
- AVXS-101
- Genetic
- Lead sponsor
- United BioSource, LLC
- Industry
- Eligibility
- Not listed
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Interventions
- Onasemnogene Abeparvovec-xioi
- Biological
- Lead sponsor
- Novartis Gene Therapies
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2035
- U.S. locations
- 18
- States / cities
- Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
- Conditions
- Spinal Muscular Atrophy, Spine Deformity, Scoliosis
- Interventions
- ThecaFlex DRx System
- Device
- Lead sponsor
- Alcyone Therapeutics, Inc
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Orange, California • Palo Alto, California + 12 more
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Risdiplam
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 1 Month to 7 Months
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2023
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Chicago, Illinois • Boston, Massachusetts + 1 more
- Conditions
- Metastatic Castration-resistant Prostate Cancer
- Interventions
- BMS-986365, Enzalutamide, Abiraterone, Docetaxel, Predinsone/Prednisolone
- Drug
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 960 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 48
- States / cities
- Birmingham, Alabama • Gilbert, Arizona • Anaheim, California + 34 more