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Showing 49–72 of 202 matching trials from the live ClinicalTrials.gov search.
Conditions
Duchenne Muscular Dystrophy, Cardiomyopathy
Interventions
Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Drug
Lead sponsor
Capricor Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Cincinnati, Ohio
Active, not recruiting Phase 1Phase 2 Interventional

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-050/NCNP-03, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 15 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 3 more
Conditions
Symptomatic Neurogenic Orthostatic Hypotension
Interventions
ampreloxetine
Drug
Lead sponsor
Theravance Biopharma
Industry
Eligibility
30 Years and older
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
13
States / cities
Colorado Springs, Colorado • Boca Raton, Florida • Port Charlotte, Florida + 10 more
Conditions
Sarcopenia
Interventions
REGN1033 (SAR391786), placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
70 Years and older
Enrollment
253 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
33
States / cities
Anniston, Alabama • Phoenix, Arizona • El Cajon, California + 30 more
Conditions
Multiple System Atrophy
Interventions
Autologous Mesenchymal Stem Cells, Placebo
Biological · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
30 Years to 70 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Hypertension
Interventions
Clonidine, Nitroglycerin transdermal, Dipyridamole/ Aspirin (Aggrenox), Desmopressin (DDAVP), Sildenafil, Nifedipine, Hydralazine, Hydrochlorothiazide, Placebo, Bosentan, Diltiazem, Eplerenone, guanfacine, L-arginine, captopril, carbidopa, losartan, metoprolol tartrate, nebivolol hydrochloride, prazosin hydrochloride, tamsulosin hydrochloride, Head-up tilt., aliskiren, Local heat stress
Drug · Dietary Supplement · Other
Lead sponsor
Vanderbilt University
Other
Eligibility
18 Years to 80 Years
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Parkinson Disease, Multiple System Atrophy, Rapid Eye Movement Sleep Behavior Disorder, Normal Pressure Hydrocephalus
Interventions
Biomarker assay
Other
Lead sponsor
Stony Brook University
Other
Eligibility
50 Years to 75 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Stony Brook, New York
Conditions
Frontotemporal Degeneration, Progressive Supranuclear Palsy, Multiple System Atrophy
Interventions
None-Observation Only
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
997 participants
Timeline
2003 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 9 more
Conditions
Autoimmune Diseases of the Nervous System, Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Disease Progression, Brain Atrophy
Interventions
Tcelna, Placebo
Biological
Lead sponsor
Opexa Therapeutics, Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
33
States / cities
Phoenix, Arizona • Tucson, Arizona • Berkeley, California + 28 more
Conditions
Parkinson Disease, Osteoporosis, Parkinsonism, Parkinson's Disease and Parkinsonism, Atypical Parkinsonism, Progressive Supranuclear Palsy, Multiple System Atrophy, Vascular Parkinsonism, Dementia With Lewy Bodies
Interventions
Zoledronic Acid 5Mg/Bag 100Ml Inj, Placebo
Drug · Other
Lead sponsor
California Pacific Medical Center Research Institute
Other
Eligibility
60 Years and older
Enrollment
2,721 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
77
States / cities
Scottsdale, Arizona • Fayetteville, Arkansas • Little Rock, Arkansas + 58 more
Completed No phase listed Observational Results available

I-Tracking Neurodegeneration in Early Wolfram Syndrome

NCT03951298
Conditions
Wolfram Syndrome
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
AVI-4658 (Eteplirsen)
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
11
States / cities
Long Beach, California • Gainesville, Florida • Chicago, Illinois + 8 more
Conditions
Oculopharyngeal Muscular Dystrophy
Interventions
Cabaletta
Drug
Lead sponsor
Bioblast Pharma Ltd.
Industry
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
2
States / cities
Los Angeles, California • Orange, California
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
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Conditions
Multiple System Atrophy (MSA)
Interventions
BHV3241, verdiperstat
Drug
Lead sponsor
Biohaven Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Morrisville, North Carolina
Conditions
Neurodegenerative Diseases, sca3, MSA - Multiple System Atrophy, Ataxia, Synucleinopathies
Interventions
18F-PBR06, 18F-PBR06
Drug · Radiation
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
50 participants
Timeline
2023 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
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
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
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Non-diabetic Neuropathy, Primary Autonomic Failure, Dopamine Beta Hydroxylase Deficiency
Interventions
Placebo, Droxidopa
Drug
Lead sponsor
Chelsea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
263 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
14
States / cities
Huntsville, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 11 more
Conditions
Hypertension, Pure Autonomic Failure, Multiple System Atrophy
Interventions
BQ123, Saline
Drug
Lead sponsor
Vanderbilt University
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
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