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:
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.