Myopathy Clinical Trials

6 recruiting

Frequently Asked Questions

Common questions about Myopathy clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 285 trials

Recruiting
Phase 1

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS)Immune-mediated Necrotizing Myopathy (IMNM)Immune Thrombocytopenia (ITP)+3 more
Chugai Pharmaceutical144 enrolled69 locationsNCT06371417
Recruiting
Phase 2Phase 3

RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

DermatomyositisIdiopathic Inflammatory Myopathy
Cabaletta Bio74 enrolled32 locationsNCT06154252
Recruiting

Characterization of Patients With Uncommon Presentations and/or Uncommon Diseases Associated With the Cardiovascular System

ObesityCardiomyopathyLi-Fraumeni Syndrome+3 more
National Heart, Lung, and Blood Institute (NHLBI)5,000 enrolled3 locationsNCT01143454
Recruiting
Phase 2Phase 3

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).

PediatricSymptomatic Obstructive Hypertrophic Cardiomyopathy
Cytokinetics55 enrolled38 locationsNCT06412666
Recruiting
Phase 1

A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy

Dilated Cardiomyopathy
AstraZeneca31 enrolled4 locationsNCT07241104
Recruiting

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

Transthyretin Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals2,000 enrolled3 locationsNCT07358078
Recruiting
Phase 3

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals1,250 enrolled157 locationsNCT07052903
Recruiting

Muscle Health Measurements Using Electrical Impedance Myography

Muscular DystrophiesMyositisCongenital Myopathy+4 more
Beth Israel Deaconess Medical Center150 enrolled1 locationNCT07502989
Recruiting
Phase 1

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Dilated Cardiomyopathy (DCM)
Rocket Pharmaceuticals Inc.8 enrolled3 locationsNCT07137338
Recruiting
Phase 3

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

CardiomyopathiesAmyloidosisHeart Diseases+3 more
Eidos Therapeutics, a BridgeBio company582 enrolled98 locationsNCT06563895
Recruiting
Phase 2Phase 3

A Study of the Efficacy and Safety of Danicamtiv in Participants With Symptomatic Genetic and Familial Dilated Cardiomyopathy

Symptomatic Genetic Dilated Cardiomyopathy
Kardigan, Inc.332 enrolled26 locationsNCT07210723
Recruiting
Phase 2

Venous Ethanol for Ventricular Tachycardia

Ventricular TachycardiaIschemic Cardiomyopathy
The Methodist Hospital Research Institute156 enrolled1 locationNCT05511246
Recruiting

Molecular and Genetic Studies of Congenital Myopathies

Nemaline MyopathyCentronuclear MyopathyCentral Core Disease+5 more
Boston Children's Hospital4,000 enrolled1 locationNCT00272883
Recruiting
Phase 3

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Intellia Therapeutics1,200 enrolled132 locationsNCT06128629
Recruiting
Phase 2

Administration of Allogeneic-MSC in Patients With Non-Ischemic Dilated Cardiomyopathy

Non-ischemic Dilated Cardiomyopathy
Joshua M Hare136 enrolled4 locationsNCT04476901
Recruiting
Not Applicable

Arrhythmogenic Activity During Exercise in ARVC Patients

Arrhythmogenic Right Ventricular Cardiomyopathy
Swiss Federal Institute of Technology120 enrolled1 locationNCT06040242
Recruiting

Natural History Study for Patients With Nemaline Myopathy in Spain

Cohort StudiesNemaline MyopathyMyopathies+13 more
Hospital Universitari Vall d'Hebron Research Institute100 enrolled1 locationNCT07488806
Recruiting
Not Applicable

Manganese-enhanced Magnetic Resonance Imaging in Takotsubo Cardiomyopathy

Takotsubo Cardiomyopathy
University of Edinburgh100 enrolled1 locationNCT07491718
Recruiting
Phase 1Phase 2

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

Hypertrophic Cardiomyopathy
Tenaya Therapeutics30 enrolled10 locationsNCT05836259
Recruiting
Phase 2

Ademetionine in Obstructive Hypertrophic Cardiomyopathy

Obstructive Hypertrophic Cardiomyopathy (oHCM)
China National Center for Cardiovascular Diseases44 enrolled3 locationsNCT07367724