- Conditions
- Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma
- Interventions
- SF1126
- Drug
- Lead sponsor
- Ezra Cohen
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016
- U.S. locations
- 1
- States / cities
- La Jolla, California
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 1129–1148
of 1,148
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Neuronal Ceroid Lipofuscinosis CLN5
- Interventions
- NGN-101
- Genetic
- Lead sponsor
- Neurogene Inc.
- Industry
- Eligibility
- 3 Years to 9 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Neurofibromatosis Type 1
- Interventions
- Not listed
- Lead sponsor
- Shriners Hospitals for Children
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Interventions
- LP352, Placebo
- Drug
- Lead sponsor
- Longboard Pharmaceuticals
- Industry
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 29
- States / cities
- Tucson, Arizona • Little Rock, Arkansas • Downey, California + 24 more
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Interventions
- VX-670, Placebo
- Drug
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 8
- States / cities
- San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
- Conditions
- Alzheimer Disease, Down Syndrome
- Interventions
- ION269
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 35 Years to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 5
- States / cities
- Indianapolis, Indiana • Kansas City, Kansas • Lexington, Kentucky + 2 more
- Conditions
- Huntington Disease, Healthy
- Interventions
- ActiGraph LEAP, Axivity AX6
- Device
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 25 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Neurofibromatosis 2
- Interventions
- Penetrating auditory brainstem implant
- Device
- Lead sponsor
- FDA Office of Orphan Products Development
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2003
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Pasadena, California • Englewood, Colorado
- Conditions
- Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
- Interventions
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Drug
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 43
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 37 more
- Conditions
- Friedreich Ataxia
- Interventions
- MR
- Device
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
- Interventions
- CLN2 Treatment
- Other
- Lead sponsor
- Jessica Scherr
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Lafora Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Tourette Syndrome, Tourette's Disorder, Chronic Motor Tic Disorder, Chronic Vocal Tic Disorder, Persistent Motor Tic Disorder, Persistent Vocal Tic Disorder, Persistent Tic Disorder, Tic Disorders
- Interventions
- mindfulness-based intervention for tics (MBIT), psychoeducation with relaxation and supportive therapy (PRST)
- Other
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Neurofibromatosis 1
- Interventions
- N-Acetyl cysteine, Placebo
- Drug · Other
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 8 Years to 16 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 1,000 participants
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
- Interventions
- Inotersen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2021
- U.S. locations
- 9
- States / cities
- Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
- Conditions
- Neuronal Ceroid Lipofuscinosis
- Interventions
- HuCNS-SC
- Biological
- Lead sponsor
- StemCells, Inc.
- Industry
- Eligibility
- 6 Months to 6 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Huntington Disease
- Interventions
- SAGE-718
- Drug
- Lead sponsor
- Supernus Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019
- U.S. locations
- 2
- States / cities
- Long Beach, California • Marlton, New Jersey
- Conditions
- Tourette Syndrome in Children, Tourette Syndrome in Adolescence
- Interventions
- T92, Placebo
- Dietary Supplement
- Lead sponsor
- Tasly Pharmaceuticals, Inc.
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut