If your child has been diagnosed with Duchenne muscular dystrophy (DMD), you may be exploring every possible option for treatment and care—including clinical trials. But how does your child participate in a Duchenne clinical trial? And where can you find trials that are actively recruiting? This guide will walk you through the steps to get involved in Duchenne research and show you trusted resources to locate clinical trials that may be right for your child.
Duchenne muscular dystrophy (DMD) is a rare and progressive genetic disorder that primarily affects boys. As science continues to search for effective treatments, clinical trials play a crucial role in advancing medical research and providing potential access to new therapies. If you’re a parent or caregiver of a child diagnosed with Duchenne, you may be wondering how your child can participate in clinical trials—and where to find them. This guide will help you understand the process and resources available.
Table of Contents
What Are Clinical Trials for Duchenne?
Clinical trials are research studies that test new cures or approaches to care. In the case of Duchenne, these trials may involve:
- Gene therapies
- Exon-skipping drugs
- Steroid alternatives
- Muscle-strengthening treatments
- Supportive therapies (e.g., cardiac or respiratory care)
Participation in a clinical trial can offer access to cutting-edge treatments before they become widely available. However, they also involve risks, so understanding the full picture is essential.
How Does My Child Participate in a Duchenne Clinical Trial?
1. Understand the Eligibility Criteria
Each clinical trial has specific inclusion and exclusion criteria. These can be based on:
- Age of the child
- Genetic mutation (specific exons involved)
- Stage of disease progression (e.g., ambulatory or non-ambulatory)
- Previous treatments or therapies
2. Speak with Your Child’s Healthcare Team
Your child’s neurologist or specialist can help determine:
- Whether your child may qualify for certain trials
- The potential benefits vs. risks
- How participation may affect ongoing care
Physicians can also refer you to clinical trial coordinators or centers with Duchenne expertise.
3. Search for Open Clinical Trials
Use clinical trial registries (see list below) to identify ongoing studies. Look for:
- Trial phase (I–IV)
- Location and sponsor (university, biotech, pharmaceutical)
- Study goals and length
- Compensation, travel support, and follow-up requirements
4. Contact the Trial Sponsor
Once you find a trial that matches your child’s profile:
- Contact the site coordinator listed in the study details
- Ask about the screening process
- Get clarity on what participation involves (testing, travel, possible side effects)
5. Enroll and Participate
If your child qualifies and you decide to move forward:
- You’ll go through a process called informed consent (and assent for older children)
- Your child will undergo initial screening tests
- Participation typically involves regular follow-up visits, data collection, and evaluations
Where Can I Find Duchenne Clinical Trials?
Finding the right clinical trial can feel overwhelming. These resources can help you search by location, age, genetic mutation, and trial phase.
🧭 Top Resources to Search Duchenne Clinical Trials
1. ClinicalTrials.gov
- Run by the U.S. National Library of Medicine
- Use keywords like “Duchenne muscular dystrophy”
- Filter by location, age, trial status (recruiting), and sponsor
2. EU Clinical Trials Register
- For families based in Europe
- Offers trials across multiple EU countries
3. myTomorrows
- AI-powered platform — a transformative tool designed to streamline clinical trial search and patient eligibility pre-screening with precision.
- In this evolution of AI platform introduce advanced automation of the eligibility checking process, delivering significantly greater time-saving efficiencies than before.
4. Parent Project Muscular Dystrophy (PPMD) – Clinical Trial Finder
- Offers a user-friendly Trial Finder Tool
- Includes detailed guides, glossary terms, and mutation-specific trial filters
5. Muscular Dystrophy Association (MDA)
- Provides research updates and clinical trial opportunities
- MDA Care Centers are often involved in clinical trials
Final Thoughts: Is a Clinical Trial Right for Your Child?
Every family’s situation is unique. Participating in a Duchenne clinical trial is a deeply personal decision that depends on many factors including health status, logistics, and emotional readiness.
Here are a few things to keep in mind:
- Ask detailed questions about what’s involved
- Balance hope with realistic expectations
- Connect with other parents in Duchenne communities or forums
- Follow DMDWarrior’s website and social media accounts
Frequently Asked Questions (FAQs)
❓ Is there a cost to participate in a clinical trial?
Most trials cover medical costs related to the study. Some may also offer travel stipends or reimbursements.
❓ What if there are no trials near me?
Some studies offer remote participation, while others help with travel logistics to trial sites.
❓ Can my child exit the trial at any time?
Yes. Participation is always voluntary, and you can withdraw your child at any time for any reason.
Take Action Today
If you’re ready to explore clinical trials for Duchenne:
✅ Talk to your child’s specialist
✅ Use trusted clinical trial databases
✅ Reach out to trial coordinators
✅ Join support networks like DMDWarrior, PPMD or MDA
Early involvement in trials can offer new opportunities for care—and contribute to the global fight against Duchenne.
