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ClinicalTrials.gov public records Last synced May 21, 2026, 10:09 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 173 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Inherited Neuromuscular Conditions Clear all
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Cancer, Skin Fibroblasts, Muscle Weakness
Interventions
Nicotinamide Riboside (NR)
Dietary Supplement
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 21, 2026, 10:09 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F-18 florbetapir PET
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 21, 2026, 10:09 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Endometriosis
Interventions
Tanezumab, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 49 Years · Female only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
30
States / cities
Mobile, Alabama • Tucson, Arizona • San Diego, California + 20 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2021 · Synced May 21, 2026, 10:09 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophy
Interventions
Placement of the BrainGate2 sensor(s) into the motor-related cortex
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2038
U.S. locations
5
States / cities
Sacramento, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced May 21, 2026, 10:09 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 10:09 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Nondystrophic Myotonias, Myotonia Congenita, Myotonic Disorders
Interventions
Not listed
Lead sponsor
Richard Barohn, MD
Other
Eligibility
6 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
4
States / cities
Kansas City, Kansas • Boston, Massachusetts • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2013 · Synced May 21, 2026, 10:09 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Arthritis, Osteoarthritis
Interventions
tanezumab, Placebo
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
697 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
82
States / cities
Phoenix, Arizona • Danbury, Connecticut • Stamford, Connecticut + 65 more
Source: ClinicalTrials.gov public record
Updated Mar 21, 2021 · Synced May 21, 2026, 10:09 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Obesity and Obesity-related Medical Conditions, Diabetes, Atherosclerotic Disease, Heart Failure, MASH, Sarcopenia, Osteoporosis, Hyperparathyroidism, Hypoparathyroidism, Ischemic Heart Disease, Cystic Fibrosis (CF), Chronic Kidney Disease(CKD), Osteopenia, Cachexia
Interventions
Endothelial cell collection
Other
Lead sponsor
Bettina Mittendorfer
Other
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2027 – 2030
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 10:09 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
Interventions
The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
Drug
Lead sponsor
Daniel Benjamin
Other
Eligibility
Up to 21 Years
Enrollment
3,520 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
43
States / cities
Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more
Source: ClinicalTrials.gov public record
Updated Sep 5, 2023 · Synced May 21, 2026, 10:09 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 30, 2025 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced May 21, 2026, 10:09 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis), Atypical Neurofibroma
Interventions
Whole Body Magnetic Resonance Imaging
Diagnostic Test
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 11, 2026 · Synced May 21, 2026, 10:09 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Breast Cancer
Interventions
suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
Drug
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
8
States / cities
San Francisco, California • Boynton Beach, Florida • Orlando, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2014 · Synced May 21, 2026, 10:09 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,323 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 10:09 PM EDT
Enrolling by invitation Not applicable Interventional
View directory record Official record
Conditions
Hypotonia, Muscle, Obstructive Sleep Apnea, Nasal Airway Obstruction
Interventions
Self-Supporting Nasopharyngeal Airway (ssNPA)
Device
Lead sponsor
University of Michigan
Other
Eligibility
3 Years to 23 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA), Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), Autoimmune Encephalitis, Celiac Disease, Celiac Disease in Children, Chronic Lyme Disease, Post-treatment Lyme Disease Syndrome, Crohn's Disease, Dysautonomia, Anorexia Nervosa, Bulimia Nervosa, ARFID, Avoidant / Restrictive Food Intake Disorder, Ehlers Danlos Syndrome, Endometriosis, Fibromyalgia (FM), Long COVID, Lupus, Migraines, Mast Cell Activation Syndrome, Multiple Sclerosis, Myalgic Encephalomyelitis (ME), Myasthenia Gravis, Generalized, Myasthenia Gravis in Children, Narcolepsy, Obsessive Compulsive Disorder (OCD), PANDAS, Pediatric Acute-onset Neuropsychiatric Syndrome (PANS), POTS - Postural Orthostatic Tachycardia Syndrome, General Anxiety Disorder, Social Anxiety Disorder, PTSD - Post Traumatic Stress Disorder, Psoriasis, Traumatic Brain Injury, Tourette's Syndrome, Inflammatory Bowel Disease (IBD), Autoimmune Diseases, Neurological Diseases or Conditions, Psychiatric Disorder, Sjogren's Syndrome, Ulcerative Colitis and Crohn's Disease
Interventions
Not listed
Lead sponsor
Brain Inflammation Collaborative
Other
Eligibility
2 Years and older
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2030
U.S. locations
1
States / cities
Delafield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 21, 2026, 10:09 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1978
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 9, 2008 · Synced May 21, 2026, 10:09 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 21, 2026, 10:09 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced May 21, 2026, 10:09 PM EDT
Suspended Phase 1 Interventional
View directory record Official record
Conditions
Prostatic Neoplasm
Interventions
EZN-4176
Drug
Lead sponsor
Enzon Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 17, 2012 · Synced May 21, 2026, 10:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
FTLD, Progressive Supranuclear Palsy (PSP), Frontotemporal Dementia (FTD), Corticobasal Degeneration (CBD), PPA Syndrome, Behavioral Variant Frontotemporal Dementia (bvFTD), Semantic Variant Primary Progressive Aphasia (svPPA), Nonfluent Variant Primary Progressive Aphasia (nfvPPA), FTD With Amyotrophic Lateral Sclerosis (FTD/ALS), Amyotrophic Lateral Sclerosis (ALS), Oligosymptomatic PSP (oPSP), Corticobasal Syndrome (CBS)
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 85 Years
Enrollment
1,489 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
15
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced May 21, 2026, 10:09 PM EDT