Considering a clinical trial

The decision to enroll your child in a clinical trial is a personal one that should be made in partnership with your child's doctor. For many families living with Duchenne, clinical trials offer a chance to receive investigational drugs, but there are many factors that need to be considered.


A clinical trial that is well-planned and well-conducted potentially offers participants the chance to:


  • Have access to investigational treatments that are being studied in research (investigational drugs)
  • Receive regular, careful medical attention from a team of healthcare professionals
  • Be part of research that may result in advancing the understanding of a disease or the approval of an investigational treatment
  • The investigational treatment may have side effects - including some very serious ones
  • The investigational treatment may not work for everyone in the trial
  • In some trials, some participants may get a placebo (a pill or liquid that has no active ingredients) instead of the drug being studied. Using a placebo gives researchers something to compare with the drug being studied. Usually, participants and researchers will not know which participants are being treated with a placebo or the drug being studied during a clinical trial
  • The routine for the investigational treatment may take more time or be more complicated than what the participant is used to. For example, participants may need to get regular tests, have frequent check-ins with the study site, stay in a hospital, or follow a plan for taking the medicine


Once you and your child's doctor have determined that a clinical trial is the right step for your family, you’ll need to find a trial that is a good match.


Each clinical study has "eligibility criteria" that must be met in order to participate. These factors determine which individuals can participate, and are different for every clinical trial. These may include:



Type and stage
of disease

Other medical

Medications or treatments participants are using or have used

Other criteria, such as a person's
health status or ability to do the
tasks needed in a study

Before your child can participate in a trial, he must go through a screening process to see if he meets these criteria. The screening process could include tests and interviews. Not everyone who applies for a clinical trial will be selected to participate.

If your child meets the eligibility criteria, the clinical trial staff will start the informed consent process. Informed consent helps you and your child decide whether or not you want to be part of a particular clinical trial.

Eligible children and their families are given the key facts about the study before it starts, including the risks and potential benefits.

The research team provides details about the study to potential participants in an informed consent document.

Informed consent explains the following:

How the study will be conducted

Specific participant requirements

Risks and benefits of participation

Study duration

Cost to you, if any

Payment to you, if any

Names and phone numbers of key contacts

If you and your child decide together that you do want to be part of the trial, you will sign the informed consent document to indicate that you understand what the study involves, and its risks and benefits. Informed consent does not mean you have to stay in the study; you can ask to leave the study at any time.

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