Considering Clinical Trials

Clinical Trial Benefits and Risks

Your patients and caregivers may be interested in finding out more about participating in clinical trials. They may view them as a way to access experimental therapies for DMD. It is important to help your patients and caregivers understand that only some patients will be selected for clinical trials and, even after being selected, not all will receive the experimental therapy. Some may receive a placebo or no therapy at all.

Below are some of the benefits and risks of clinical trials for your patient to consider:


  • Potential access to investigational treatments being studied in research
  • Regularly scheduled medical attention from a team of healthcare professionals
  • Participating in research that may result in advancing the understanding and treatment of the disease
  • Playing a part in the approval of an investigational treatment


  • The investigational treatment may have side effects, including some serious ones
  • Some participants may get a placebo (a pill or liquid that has no active ingredients) instead of the drug being studied
  • The procedure for the investigational treatment may take more time or be more complicated than routine treatments. Participants may need regular testing, frequent check-ins, or a hospital stay
  • The investigational treatment may not work


Your patients and caregivers may be wondering whether a clinical trial could be right for them. Speak with your patients and caregivers about suitable clinical trial options.

What Your Patients and Caregivers Could Expect if Selected for a Clinical Trial

If your patient is selected for a clinical trial, your patient will need to provide informed consent. As part of the informed consent process, the trial team will explain several aspects of the trial to your patient. Explain to your patient and caregiver that they should take time to understand this information and ask questions if they need to.

Informed consent explains the following:

  • How the study will be conducted
  • Risks and benefits of participation
  • Cost to your patient, if any
  • Names and phone numbers of key contacts
  • Specific participant requirements
  • Study duration
  • Payment to your patient, if any

Some things to consider when you read about the clinical trial:


  • Duration of the trial: Some trials are short, while others can last for years
  • Travel: Your patient and their caregiver may need to travel or stay in hospitals for tests and/or treatments


  • How many, how often, and what type of samples will be taken
  • How often your patient’s treatment team (doctors, nurses, social workers, and other healthcare providers) will monitor or administer tests to your patient
  • Which types of data will be collected and how they will be stored
  • Whether the treatment team will ask your patient or their caregiver to do other tasks, such as keeping a health log or filling out forms about how your patient feels


  • Possibility of getting placebo instead of the experimental treatment, and your patient may not know which they are receiving
  • Possibility of treatment not working
  • Possible side effects

As children with DMD get older and their disease progresses, their scores on the tests used in clinical trials may get worse. These tests may include ones that measure strength and endurance, as well as the ability to perform activities of daily living. Reassuring your patient that this is to be expected is very important and may help alleviate negative emotions.

For patients who are not eligible for current clinical trials, you may want to discuss other treatment possibilities.

Treatment options and clinical trial eligibility are continually evolving.

A number of community resources are available to help support your patients who are considering clinical trials.

Drug Development and FDA Approval

Find out more about the steps involved in the development and FDA approval of new drugs.