Exploring clinical trials for Duchenne muscular dystrophy

Clinical trials are research studies that evaluate new treatments to see if they are safe and effective. These treatments are experimental and not yet approved for regular use. Clinical trials help doctors better understand medical conditions and find new ways to treat them. They follow strict rules and are reviewed by independent ethics committees to protect the safety of participants.

Families consider clinical trials for different reasons. Some want access to treatments not yet available outside of research, especially if approved treatments are not available for their child's specific mutation or if these are not working well. Other families value the regular check-ups and care from a specialized Duchenne clinical trial teams or want to contribute to Duchenne research to help future generations.

Like any medical treatment, clinical trials involve risks, including potential side effects from the investigational treatment. Clinical trials therefore include extra tests and clinic visits so the team can closely monitor your child’s health. Each trial is reviewed by an independent ethics committee before it begins. The clinical trial team will explain any known risks before joining a trial and participants can withdraw at any time.

Duchenne trials follow strict safety protocols designed with Duchenne-specific considerations, but like any medical treatment, they involve risk. Research teams experienced in Duchenne understand participants' unique medical needs and disease progression. Before enrolling, the clinical trial team will carefully evaluate whether the trial is appropriate for your child's current health status. Your child's safety is monitored closely throughout the clinical trial.

No. Before a treatment can be tested in children living with Duchenne muscular dystrophy, researchers must demonstrate that it has shown potential benefit and is safe to use. All trials follow strict ethical guidelines that require informed consent, meaning you will fully understand what participation involves before deciding. You and your child (if age-appropriate) have the right to ask questions, consult with your regular doctors, and take time to make your decision. Independent ethics committees review every trial to ensure children are protected. Participation is always voluntary, and you can withdraw at any time.

Some Duchenne trials use placebos to accurately measure if the investigational treatment is effective. If a trial includes a placebo, you will be told upfront, and the clinical trial team will explain whether your child might receive the placebo or investigative treatment (assignment is often random).  In cases where a placebo is being used, you will not know whether your child has received the investigational treatment or a placebo. Clinical trials for progressive conditions like Duchenne are carefully designed to lower risks for those receiving placebo. All children receive regular check-ins and care regardless of whether they receive the placebo or the investigational treatment. Some trials allow children to continue their standard treatments (like corticosteroids). The clinical trial team will explain the specific design of the trial you are considering.

Every clinical trial has a set of rules, called eligibility criteria, that determine who can join. These may include age, diagnosis, medical history, or current treatments. Eligibility criteria help make sure the trial’s results are accurate and aim to protect the safety of the children who join the clinical trial. For Duchenne, many clinical trials are mutation-specific. This means they only accept children with certain genetic deletions or duplications. Your patient navigator will review the eligibility criteria of Duchenne trials and help review which trials might be relevant for your child's specific situation.

During your call, your patient navigator will ask questions to collect the information needed to look for relevant trial options. After reviewing this information, your navigator will share any trial options that may be a match. You can discuss these options with your doctor to help you make a decision about whether to partake in a clinical trial. If you are interested in learning more about a trial, your patient navigator can connect you with the clinical trial team, who will explain the trial’s details and discuss next steps to see if participation may be possible. There is no obligation to join a trial, and you can take the time you need to consult with your doctor and decide what feels right for your family.

Yes. myTomorrows supports many families affected by Duchenne that live outside the U.S. If you are interested in a clinical trial happening in a different country, your navigator can explain what participation would involve, including travel, visas, and site logistics. Some studies may cover certain travel-related costs, while in other cases families may need to arrange their own travel.