Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Duchenne Muscular Dystrophy Clinical Trials

A clinical trial is a test in humans to evaluate biomedical or health-related outcomes, including experimental medications or therapies. Clinical trials are experiments, not treatments, and participation requires careful consideration.

Although it's possible to benefit from participating in a clinical trial, it's also possible that no benefit — or even harm — may occur. Keep your MDA clinic doctor informed about any clinical trial participation. (Note that MDA has no ability to influence who is chosen to participate in a clinical trial.)

Clinical trials are conducted in a series of successive steps, called phases. Each phase is designed to answer a separate research question.

  • Observational: Tests health or biomedical outcomes to understand the disease course. These studies are conducted without an intervention (e.g., an investigational new drug).
  • Phase 0: Tests how a new drug distributes in the body at a very low dose (below what is expected to have a therapeutic benefit).
  • Phase 1: Tests whether a new drug is safe in a small group of people, and determines the best way to dose it.
  • Phase 2: Tests whether a new drug is effective in a medium-sized group of people, and confirms its safety.
  • Phase 3: Tests whether a new drug is effective in a large group of people, and monitors its side effects and safety.
  • Phase 4: Studies the drug after it has been marketed to understand the benefits and side effects associated with long-term use.

*Note: Some clinical trials are not assigned a phase by the U.S. Food and Drug Administration (FDA). These include trials that test devices or behavioral interventions.

Duchenne Muscular Dystrophy (DMD) Clinical Trials

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

Status: 
Recruiting
Phase: 
Phase 1
Phase 2

Treatment Effect of Tamoxifen on Patients With DMD

Status: 
Not Yet Recruiting
Phase: 
Phase 1

Rimeporide in Patients With Duchenne Muscular Dystrophy

Status: 
Recruiting
Phase: 
Phase 1

Study of SRP-4045 and SRP-4053 in DMD Patients

Status: 
Not Yet Recruiting
Phase: 
Phase 3

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

Status: 
Recruiting
Phase: 
Phase 1
Phase 2

Stacking Exercises Aid the Decline in FVC and Sick Time

Status: 
Recruiting
Phase: 
Phase 4

Pediatric Radio Frequency Coils Generic

Status: 
Recruiting
Phase: 
Phase 1
Phase 2

Stem Cell Therapy in Duchenne Muscular Dystrophy

Status: 
Recruiting
Phase: 
Phase 1

DuchenneConnect Registry

Status: 
Recruiting

Sodium Nitrate for Muscular Dystrophy

Status: 
Recruiting
Phase: 
Phase 1

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Status: 
Recruiting
Phase: 
Phase 1
Phase 2

Prospective Becker-Heart-Study

Status: 
Recruiting

Confirmatory Study of Eteplirsen in DMD Patients

Status: 
Recruiting
Phase: 
Phase 3

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