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Showing 1–24 of 895 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neuromuscular; Disorder, Hereditary Clear all
Completed No phase listed Observational

Carpal Tunnel Syndrome and Amyloid Cardiomyopathy

NCT02792790
View directory record Official record
Conditions
Amyloidosis, Cardiomyopathy, Transthyretin Amyloidosis, Senile Systemic Amyloidosis, Carpal Tunnel Syndrome
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
50 Years and older
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced Jun 27, 2026, 2:26 AM EDT
Terminated No phase listed Observational

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435
View directory record Official record
Conditions
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
11 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Jun 27, 2026, 2:26 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04860362
View directory record Official record
Conditions
Neurofibromatosis 1, Social Skills
Interventions
PEERS
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
12 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 25, 2021 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 1 Interventional

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

NCT02295748
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
4 Years and older · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Jun 27, 2026, 2:26 AM EDT
Recruiting Phase 1Phase 2 Interventional

Sapanisertib and Serabelisib (PIKTOR) in Various Combinations in Patients With HR+/HER2- Advanced/Metastatic Breast Cancer

NCT07558733
View directory record Official record
Conditions
HR+ HER2- Breast Cancer
Interventions
Serabelisib, Sapanisertib, Fulvestrant
Drug
Lead sponsor
Faeth Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
3
States / cities
Los Angeles, California • Springfield, Oregon • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Jun 27, 2026, 2:26 AM EDT
Completed No phase listed Observational

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

NCT01437345
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
7
States / cities
Sacramento, California • Washington D.C., District of Columbia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 10, 2017 · Synced Jun 27, 2026, 2:26 AM EDT
Recruiting No phase listed Observational

The Myelin Disorders Biorepository Project

NCT03047369
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 Jun 27, 2026, 2:26 AM EDT
Completed Phase 2 Interventional Results available

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

NCT06304064
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 23, 2024 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 2 Interventional

Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease

NCT00876863
View directory record Official record
Conditions
Alzheimer's Disease
Interventions
CERE-110: Adeno-Associated Virus injection Surgery, Placebo Surgery
Drug · Procedure
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
55 Years to 80 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2020 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 29, 2020 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 2 Interventional Results available

Dosing Study of Replagal in Patients With Fabry Disease

NCT00068107
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced Jun 27, 2026, 2:26 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Magnetic Stimulation of the Human Nervous System

NCT00001780
View directory record Official record
Conditions
Demyelinating Disease, Healthy, Lysosomal Storage Disease, Motor Neuron Disease, Movement Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jun 27, 2026, 2:26 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Neuromuscular Characteristics of Individuals With Down Syndrome (DS)

NCT00643955
View directory record Official record
Conditions
Down Syndrome
Interventions
muscle strength
Other
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
18 Years to 45 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2008
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced Jun 27, 2026, 2:26 AM EDT
Enrolling by invitation Phase 4 Interventional

N-of-1 for Beta-Blockers in Cardiac Amyloidosis

NCT05019027
View directory record Official record
Conditions
Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions
Beta blocker
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
65 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Jun 27, 2026, 2:26 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer

NCT03437941
View directory record Official record
Conditions
Metastatic Castration-Resistant Prostate Cancer
Interventions
Exicorilant, Enzalutamide, Placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Scottsdale, Arizona • Detroit, Michigan • Basking Ridge, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 3 Interventional

High-dose Prednisone in Duchenne Muscular Dystrophy

NCT00110669
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 10 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced Jun 27, 2026, 2:26 AM EDT
Recruiting Phase 2 Interventional

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

NCT05985161
View directory record Official record
Conditions
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1 Gene Mutation, Solid Tumor
Interventions
Selinexor
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
12 Months and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Not applicable Interventional

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

NCT02654886
View directory record Official record
Conditions
Pompe Disease, Muscle Weakness
Interventions
Resistance Exercise Training
Other
Lead sponsor
University of California, Irvine
Other
Eligibility
16 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Sep 25, 2019 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect

NCT03627494
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
GSK3439171A, Placebo
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced Jun 27, 2026, 2:26 AM EDT
Recruiting No phase listed Observational

Congenital Muscle Disease Study of Patient and Family Reported Medical Information

NCT01403402
View directory record Official record
Conditions
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
Interventions
Not listed
Lead sponsor
Cure CMD
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Lakewood, California
Source: ClinicalTrials.gov public record
Updated Aug 8, 2021 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 1 Interventional

Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

NCT02415153
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Primary Central Nervous System Neoplasm, Refractory Primary Central Nervous System Neoplasm
Interventions
Laboratory Biomarker Analysis, Pharmacological Study, Pomalidomide
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 20 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
12
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Jun 27, 2026, 2:26 AM EDT
Completed Phase 1 Interventional

Sickle Cell Pain: Intervention With Capsaicin Exposure

NCT03899246
View directory record Official record
Conditions
Neuropathic Pain, Sickle Cell Disease
Interventions
Eight Percent Topical Capsaicin
Drug
Lead sponsor
Children's Hospital of Michigan
Other
Eligibility
14 Years to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 28, 2020 · Synced Jun 27, 2026, 2:26 AM EDT
Recruiting Phase 1 Interventional

A Study of AZD2962, an IRAK4 Inhibitor (IRAK4 [a Body Protein] Blocker), in Participants With Haematologic Neoplasms (Blood Cancers)

NCT07064122
View directory record Official record
Conditions
Haematologic Neoplasms
Interventions
AZD2962
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 110 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Miami, Florida • Tampa, Florida • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jun 27, 2026, 2:26 AM EDT