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Questions About Duchenne Muscular Dystrophy Clinical Trials

What to ask your child’s healthcare providers about new treatments and clinical trials for Duchenne muscular dystrophy.

Participating in a clinical trial for an investigational treatment for DMD can be a big decision for a family, but it may have several benefits.

Duchenne muscular dystrophy (DMD) is a genetic disorder that causes the progressive loss of muscle strength and muscle mass, and eventually leads to difficulty with movement, breathing, and heart function. There is currently no known cure for DMD, and treatment focuses on slowing the progression of the disease, addressing symptoms and complications, and improving the quality of life for people who have DMD.

Numerous therapies are in development for DMD, including gene therapies and exon-skipping drugs. Clinical trials are a vital part of the development process for these treatments, and if you are the parent or caregiver of a child living with DMD, clinical trials may offer your child access to treatments that are not yet available.

What are the benefits and risks?

Clinical trials are research studies that test new therapies to see if they are safe and effective. Participating in a clinical trial for an investigational treatment for DMD can be a big decision for a family, but it may have several benefits:

  • Access to potentially effective therapies that are not yet available to the general public.
  • The opportunity to contribute to medical research and help advance the development of new therapies for DMD.
  • Access to a team of healthcare professionals who are specialized in DMD and who will closely monitor a participant’s health and progress throughout the trial.
  • Being a part of a clinical trial may also help reduce the cost of treatment, as medical care and checkups may be included.

While there are potential benefits, there are also potential drawbacks, and both should be discussed with your child’s healthcare team when you are considering applying to a clinical trial.

What should you ask a healthcare provider?

Each clinical trial is different. Some may be a good fit for a particular person, and others may not. A good first question for your child’s healthcare team is “What clinical trials are available?” You can also check clinicaltrials.gov.

If you have a specific clinical trial in mind, here are some questions to discuss with your child’s healthcare providers:

  • What is the therapy being tested, and how does it work?
  • What are the potential benefits and risks of participating? Is there a risk of side effects?
  • Is there the possibility that a person in this trial will receive a placebo instead of the investigational therapy?
  • What makes a person eligible for this trial, and is my child eligible?
  • What is the phase of this clinical trial? What are the phases of clinical trials and what do they mean?
  • Have there been other clinical trials with this therapy or similar therapies? Are the results published?
  • Where is the clinical trial being conducted? How much travel is involved? Are there any out-of-pocket costs for my family?
  • What is the duration of the clinical trial?
  • What tests will be performed during the clinical trial?
  • How will my child's health be monitored, and what happens if there are any complications or side effects?
  • Will my child still receive standard-of-care treatments for DMD during the clinical trial?
  • What happens after the clinical trial ends, and will my child be able to continue receiving the investigational therapy if it is effective?
  • Can participating in this clinical trial impact my child’s health in any other ways?
  • What steps will be taken to protect my child’s health and personal information during the clinical trial?

In addition to these questions, parents and caregivers should also discuss the ways that participating in a clinical trial can impact a child's daily life and routines, as well as any logistical considerations such as travel to and from appointments and time off work or school.

Article sources open article sources

National Organization for Rare Disorders. Duchenne Muscular Dystrophy.
MedlinePlus. Duchenne muscular dystrophy.
NIH Genetic and Rare Diseases Information Center. Duchenne muscular dystrophy.
Vijay Venugopal and Steven Pavlakis. Duchenne Muscular Dystrophy. StatPearls. July 11, 2022.
Children's Hospital of Philadelphia. Gene Therapy for Duchenne Muscular Dystrophy.
Parent Project Muscular Dystrophy. Drug Development Pipeline.
MedlinePlus. Clinical Trials.
National Institute on Aging. Clinical Trials: Benefits, Risks, and Safety.
National Cancer Institute. Questions to Ask Your Doctor about Treatment Clinical Trials.
University of Virginia School of Medicine. Clinical Research: Questions You Should Ask.

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