How to Find Duchenne Muscular Dystrophy Clinical Trials Your Child May Be Eligible For

Introduction

Looking into Duchenne clinical trials for your child who has been diagnosed can raise a lot of questions. You may be trying to understand which options could be relevant, what taking part could involve, and how a trial might fit with your child’s care and your family’s day-to-day life.

Duchenne affects about 1 in 3,500 to 5,000 live male births¹ worldwide, but the details that matter for a clinical trial can be very specific to your child. Their exact genetic information, age, walking ability, heart and lung health, past and current treatments, and location may all affect which trials are worth exploring.

This guide will help you work through those questions step by step. It covers what information to have ready, where to look for Duchenne clinical trials, how to tell whether a trial may be relevant to your child, and what to consider before deciding whether to explore it further with their care team.

Key takeaways

• Your child’s exact dystrophin gene change, or mutation, and its location in the gene (exons), can help narrow the search for Duchenne clinical trials
• There are several ways to look for Duchenne clinical trials. Public online clinical trial registries, specific resources such as The Duchenne Registry and the DMD Hub Clinical Trial, and myTomorrows can all help families and caregivers find and review possible trials.
• Your child’s age, walking ability, past or current treatments, and location can all affect which trials may apply to your child.
• A trial marked “recruiting” may not be open at every trial site. Check the status of the site your family may need to visit.
• Before exploring a trial further, consider the available study data, what your child may experience, possible benefits and risks, future treatment options, costs, and the support available.
• Finding a possible match starts a process rather than ending the search. Pre-screening, referral, and site screening may still follow before enrollment is considered.
• A patient navigator can help you understand trial information and support a referral if you and your child’s physician choose to explore a trial. The site team still decides whether the trial is a good fit for your child and whether they meet the requirements to participate.

How can I find Duchenne clinical trials that may be relevant for my child?

If you are looking into Duchenne clinical trials for your child, you may be trying to understand whether there are options that could fit their needs and situation. Because every child with Duchenne is different, a trial that looks relevant at first may not be the right fit once you learn more. These resources can help you see what Duchenne clinical trials are available today:

• ClinicalTrials.gov for studies in the United States and other countries
• The Duchenne Registry from Parent Project Muscular Dystrophy
• DMD Hub Clinical Trial Finder for Duchenne studies in the UK
• World Duchenne Organization for a broader view of Duchenne research around the world
• myTomorrows brings together trial data from ClinicalTrials.gov, (Clinical Trials Information System (CTIS) and International Standard Randomised Controlled Trial Number (ISRCTN, with patient navigation support.

The next step is to look at the details that are specific to your child. Having that information ready can help you and their care team understand which trials may be worth exploring further.

How can I make sense of Duchenne trial options for my child?

When you are trying to understand what trial options may be possible for your child, there can be a lot of information to work through. Families may also have different amounts of time or experience when it comes to following Duchenne research. Different sources can help in different ways:

• Public clinical trial registries, such as ClinicalTrials.gov can show you a wide range of trials. But a trial listing may not tell you what the study could mean for your child or family.
• Duchenne organizations and patient advocacy groups can share information about research and trials that are focused on Duchenne.
• Patient navigation support gives you someone qualified to discuss all the relevant information. At myTomorrows, patient navigators use trial information from several public registries along with details about your child. They can help identify options that may be relevant and explain what may happen next.

Your child’s care team can also help you think about a trial based on their health, treatment, and daily care. If you decide to look into a trial further, the trial site will still need to review your child’s information before deciding on next steps.

What information should I have ready before starting my search?

Before you look more closely at a trial, it can help to have a few key details about your child nearby. These details can give you a better sense of whether a study may be worth asking about.

• Genetic test results: Your child’s genetic information, including their exact dystrophin gene mutation and its location in the gene (exons), is often important because some Duchenne trials focus on certain mutations in specific parts of the gene, while other mutations may exclude a child from taking part.
• Age: Duchenne trials may have different age requirements. Many are open only to children of a certain age, while others may include older age groups.
• Movement: Some trials are for children who can walk, while others may include children who cannot.
• Current and past treatments: The medicines your child takes now and treatments they had tried before may matter.
• Heart and lung health: Some trials have certain heart or breathing requirements.
• Recent medical information: Test results or notes from your child’s care team may be useful when you look at a trial in more detail.
• Location: Where you live can affect which trial sites your family may be able to reach.

It is natural for the trial search to carry hope as well as uncertainty. You do not need to understand every detail on your own. Your child’s care team can help explain their medical information, and a patient navigator can help you understand how it may relate to possible trial options.

Is the trial currently recruiting and accepting new participants?

Once you have the information that may matter to your child, you may find a trial you want to ask about. Before your family starts thinking about what taking part could mean, it helps to know whether a site is accepting new participants.

Clinical trials use different terms to show whether they are open:

If you need help understanding which trials are open and may be worth asking about, a myTomorrows patient navigator can help you review the information and explain what the next steps may involve.

What would taking part in this trial involve for my child and our family?

Even if a trial may be an option for your child, it is worth thinking about what taking part could mean for daily life. Some trials may involve regular visits over many months or years, extra tests, or time away from home. Things to think about include:

• Where the trial site is: Your family may need to travel, sometimes more than once.
• How often visits are needed: Visit frequency may vary during screening, treatment, and follow-up. Visits may include lab tests, physical or occupational therapy, and other assessments, such as EKGs or echocardiograms. You can also ask whether any visits can be completed remotely instead of at the trial site.
• How the trial fits with school and family life: Visits may mean time away from school, work or other responsibilities.
• How it fits with your child’s usual care: It is important to understand how trial visits and treatment may fit your child’s regular Duchenne care.
• Travel and concierge support: Some trials may offer help with booking travel, arranging hotels or transportation, and covering or reimbursing certain related costs. This kind of support can reduce the amount of planning families have to manage themselves and make it easier to understand what taking part would require.

Travel and trial visits can affect school, work, family routines, and your child’s regular care. Some trials may offer concierge-style support with travel planning, bookings, or reimbursement. Knowing what help is available early can give your family a better sense of what taking part would involve and what you would need to manage on your own.

What should I ask before deciding whether to explore a trial further?

Before deciding whether to explore a trial further, it can help to think about more than whether your child may be able to take part. The questions below can help you understand what the trial could mean for your child, family, and the choices you may have later.

Depending on your child’s age, they may also be asked to give their assent before taking part in a trial. This gives the trial an opportunity to explain what participation would involve in a language your child can understand and to give them time to ask questions. Parents and caregivers can also help talk through the information with their child.

For Terri Ellsworth, whose son Billy was diagnosed with Duchenne at age four and later spent more than five years in a clinical trial, these questions are personal. As a parent, she had to think not only about the trial, but also about what taking part would mean for Billy and their family.

“Participating in a clinical trial takes courage, time, sacrifice, and commitment,” shared Ellsworth.

What happens after I find a Duchenne clinical trial I want to explore?

After you have spent time thinking about whether a trial may be worth exploring for your child, you may wonder what happens next. You and your child may move through several steps before you know whether taking part could be an option. There may also be periods of waiting between these steps, and trial timelines can change.

• Step 1: Talk it through
  • You may want to discuss the trial with your child’s physician, a patient navigator, or the trial team. This is a chance to ask questions and understand what the trial could mean for your child.
• Step 2: Share some information about your child
  • Basic details about your child, such as their diagnosis, genetic information, age, health, and treatment history may be reviewed. This is sometimes called pre-screening. It can help show whether it makes sense to explore the trial further, but it does not confirm eligibility.
• Step 3: Connect with the trial site
  • If you want to take the next step, a patient navigator may be able to help with a referral. With your consent, relevant health information can be shared with the site for review.
• Step 4: The site may ask for more information
  • The trial team may request medical records, tests, or other health information. They may also schedule a screening visit to see whether your child meets the full study requirements.
• Step 5: Decide what feels right for your child and family
  • If your child is found to be eligible, the trial team will explain what taking part would involve. You may also be asked to review consent forms, and depending on their age, your child be asked for their assent. Take time to ask about anything that is unclear before deciding whether to move forward.

Exploring a trial does not mean you are committing your child to take part. The site decides whether the trial is suitable for your child and whether they are eligible. You and your family will decide whether participation is something you want to pursue.

How can a patient navigator help?

When you are looking into clinical trials for your child, there can be a lot of information to work through. A patient navigator gives you one person to come back to as you try to understand what may be possible for your child.

At myTomorrows, patient navigators combine healthcare knowledge with experience supporting families through the clinical trial process. They can help explain complex information in plain language and give families one person to come back to as questions come up. With navigators working across different regions, time zones, and languages, families can access support from a team built to work across different healthcare settings and needs.

The process starts with a conversation about your child. Your patient navigator will ask about their diagnosis, health, and past treatments. The information you share is de-identified before it is used to search for trial information from several public registries.

Your navigator can then talk you through the results and help you understand which trials may be relevant based on the information you shared. They can explain what the trial involves and what may happen next.

“One of the most meaningful parts of my work is helping families feel more grounded when the clinical trial search starts to feel overwhelming. I spend a lot of time explaining what the information means, answering questions, and walking families through each step so they know what to expect,” shared Madeleine Pagel, Expert Patient Navigator at myTomorrows, “I want patients and their families to feel they have someone in their corner as they work through their options.”

If you and your child’s physician decide to explore a trial further, your patient navigator can also help with a referral to the trial site. With your consent, the health information needed for the referral can be shared securely with the site for review.

Patient navigation works alongside your child’s care team. A navigator does not give medical advice or decide whether your child can take part in a trial. Your child’s physician continues to guide their medical care, and the trial site decides whether further evaluations are needed to determine your child’s eligibility for the trial.

Taking the next step with Duchenne clinical trials

Looking into Duchenne clinical trials may involve many different questions. You may need to understand what trials are available, what information about your child may matter, whether a trial is recruiting, and what taking part could mean for everyday life.

You may also be thinking beyond the trial itself. Possible benefits and risks, your child’s comfort, future treatment options, costs, and support available can all be part of the decision.

You do not have to work through every part of this on your own. Your child’s physician can help you think about a trial alongside their health and current care. A patient navigator can help you understand the trial information and possible next steps, while the trial team can answer questions and determine whether your child is eligible.

Taking the next step can simply mean learning more and asking the questions that matter most for your child and family.

Frequently asked questions about Duchenne clinical trials

Do I need to speak with my child’s doctor before we explore a trial?

You can learn about Duchenne clinical trials without a referral. But if a trial seems like something you may want to consider for your child, their physician can help you understand how it may fit with their health, current treatment, and care.

Can my child take part in a Duchenne clinical trial in another country?

Maybe. It depends on the trial and the site. Some sites may consider children who live in another country, while others may have rules about where people must live. For a family, the bigger questions may be what travel would involve, how often your child would need to be away from home, and what support may be available. The trial team can explain what would be required and whether travel or reimbursement support is offered.

Helpful resources

• Genetic Testing for Duchenne Muscular Dystrophy
• Duchenne Muscular Dystrophy Mutation Types
• Duchenne Muscular Dystrophy and CRISPR Technology
• Gene Therapy for Duchenne Muscular Dystrophy
• A Guide to Duchenne Muscular Dystrophy Treatments and Ongoing Research

Navigate clinical trial possibilities with myTomorrows

myTomorrows is a global health technology company that connects patients, physicians, trial sites, and BioPharma partners to enable earlier and better access to pre-approval treatments. Through a centralized, multi-stakeholder platform, myTomorrows combines AI-driven insights with human expertise to deliver tailored solutions that help overcome some of today’s greatest barriers to patient access. By simplifying and bringing transparency to the path of discovery and access to treatment, myTomorrows accelerates global patient access while supporting more efficient drug development.

Headquartered in Amsterdam, with an office in New York City, myTomorrows has supported more than 17,700 patients and 4,000 physicians across 1,777+ sites in over 144 countries and partners with 86+ of the world’s leading pharmaceutical companies.