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ClinicalTrials.gov public records Last synced May 21, 2026, 11:09 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Inherited Neuromuscular Conditions Clear all
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Cancer, Pancreatic Cancer, Sarcoma, Renal Cancer, Refractory Cancer, Refractory Neoplasm, Refractory Non-Hodgkin Lymphoma, Pancreatic Adenocarcinoma, Resistant Cancer, Neoplasm Metastasis, Neoplasm of Bone, Neoplasm, Breast, Neoplasm of Lung, Neoplasms,Colorectal, Neoplasms Pancreatic, Malignant Glioma, Malignancies, Malignancies Multiple, Bone Metastases, Bone Neoplasm, Bone Cancer, Pancreas Cancer, Pancreatic Neoplasms, Breast Neoplasms, Acute T Cell Leukemia Lymphoma
Interventions
9-ING-41, Gemcitabine - 21 day cycle, Doxorubicin., Lomustine, Carboplatin., Nab paclitaxel., Paclitaxel., Gemcitabine - 28 day cycle, Irinotecan
Drug
Lead sponsor
Actuate Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
40
States / cities
Phoenix, Arizona • Tucson, Arizona • Orange, California + 34 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced May 21, 2026, 11:09 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 64 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced May 21, 2026, 11:09 PM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 21, 2026, 11:09 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle, Dyskinetic Syndrome, Epilepsy, Glaucoma, Huntington Disease, Inflammatory Bowel Disease (IBD), Multiple Sclerosis, Neuropathy, Opioid Use, Parkinson Disease, HIV/AIDS, Ptsd, Intractable Pain, Sickle Cell Disease, Terminal Illness
Interventions
Registry, PK microsampling of blood
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced May 21, 2026, 11:09 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Albuterol, Oxandrolone
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced May 21, 2026, 11:09 PM EDT
Not listed Phase 3 Interventional
View directory record Official record
Conditions
Overactive Bladder
Interventions
onaBoNT-A, Oxybutynin ER
Drug
Lead sponsor
Christopher Patrick Smith
Other
Eligibility
18 Years to 80 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 29, 2016 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2015 · Synced May 21, 2026, 11:09 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Nausea, Vomiting, Familial Dysautonomia
Interventions
Dronabinol, Placebo
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 60 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 12, 2019 · Synced May 21, 2026, 11:09 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Neurofibromatosis 1, Chronic Pain
Interventions
iCanCope, iCanCope+Contingency Management
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 72 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced May 21, 2026, 11:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 11:09 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced May 21, 2026, 11:09 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Multiple Sclerosis, Spinal Cord Injury, Chronic Pain, Other and Unspecified Amputation Stump Complications, Muscular Dystrophies, Low Back Pain
Interventions
Pain Self-Management Intervention #1, Pain Self-Management Intervention #2, Pain Self-Management Intervention #3, Pain Self-Management Intervention #4
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 16, 2018 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2023 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Autosomal Dominant Hypocalcemia
Interventions
Not listed
Lead sponsor
Calcilytix Therapeutics, Inc., a BridgeBio company
Industry
Eligibility
Up to 90 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
9
States / cities
Oakland, California • Aurora, Colorado • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced May 21, 2026, 11:09 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced May 21, 2026, 11:09 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Chronic Mental Illness, Chronic Physical Illness
Interventions
Patient-Directed Wellness Account, Health Navigator
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
21 Years to 55 Years
Enrollment
1,663 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 2, 2017 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
Interventions
Lovastatin, reading tutoring intervention, Placebo Oral Tablet, Other Academic "sham" tutoring
Drug · Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 20 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 23, 2025 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting Early Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Ehlers-Danlos Syndrome (EDS), Complex Regional Pain Syndrome (CRPS), Amplified Musculoskeletal Pain Syndrome (AMPS), Low Back Pain, Fibromyalgia, Chronic Widespread Pain
Interventions
Two-point discrimination training, One-point discrimination training
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
10 Years to 17 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2027
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 5, 2025 · Synced May 21, 2026, 11:09 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions
RAY121
Drug
Lead sponsor
Chugai Pharmaceutical
Industry
Eligibility
18 Years to 85 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
11
States / cities
Orange, California • Baltimore, Maryland • Lake Success, New York + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced May 21, 2026, 11:09 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions
BFB-101 (AAV9-CBh-AP4B1)
Biological
Lead sponsor
BlackfinBio Ltd
Industry
Eligibility
12 Months to 60 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 27, 2025 · Synced May 21, 2026, 11:09 PM EDT
Active, not recruiting Not applicable Interventional
View directory record Official record
Conditions
Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida, Development Delay, Genetic Disease
Interventions
Power Wheelchair Standing Device
Device
Lead sponsor
Grand Valley State University
Other
Eligibility
5 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced May 21, 2026, 11:09 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced May 21, 2026, 11:09 PM EDT
No Longer Available No phase listed Expanded access
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Pridopidine
Drug
Lead sponsor
Prilenia
Industry
Eligibility
18 Years and older
U.S. locations
31
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 26 more
Source: ClinicalTrials.gov public record
Updated May 17, 2025 · Synced May 21, 2026, 11:09 PM EDT
Not yet recruiting No phase listed Observational
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
Interventions
NT3 blood draw
Diagnostic Test
Lead sponsor
Zarife Sahenk
Other
Eligibility
7 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 23, 2025 · Synced May 21, 2026, 11:09 PM EDT