High-Dose Eteplirsen for DMD
(MIS51ON Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests higher doses of eteplirsen, a treatment designed to slow the progression of Duchenne muscular dystrophy (DMD), a genetic disorder causing muscle weakness. It examines whether doses of 100 mg/kg and 200 mg/kg are safe and more effective than the standard 30 mg/kg dose. The trial is suitable for males diagnosed with DMD who have a specific genetic mutation treatable by skipping exon 51, can walk independently, and have been on a stable dose of corticosteroids. Researchers will monitor participants for safety and effectiveness over time. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment option.
Will I have to stop taking my current medications?
The trial requires that you stop taking any medications other than corticosteroids for at least 12 weeks before joining. If you're on corticosteroids, you must have been on a stable dose for at least 12 weeks before starting the trial.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that eteplirsen is generally safe for treating Duchenne muscular dystrophy (DMD). The FDA has approved it for patients who can benefit from a specific genetic treatment called exon 51 skipping. Previous studies found that eteplirsen is well-tolerated by patients, including young boys aged 6 to 48 months.
Thorough safety checks, including a study examining its long-term safety over four years, consistently show that eteplirsen is safe and well-tolerated. While some side effects can occur, data suggest they are manageable and not serious.
Prospective clinical trial participants should discuss the possible risks and benefits with their healthcare provider.12345Why do researchers think this study treatment might be promising for DMD?
Unlike the standard treatments for Duchenne muscular dystrophy (DMD), which often focus on managing symptoms and slowing progression, eteplirsen is designed to directly target the genetic cause of the disease. Eteplirsen employs exon skipping, a technique that helps the body produce a functional version of dystrophin, the protein lacking in DMD patients. Researchers are excited because this method offers a more targeted approach, potentially leading to improved muscle function and slower disease progression compared to existing therapies. Each dose variation of eteplirsen in the trial—ranging from 30 mg/kg to 200 mg/kg—allows researchers to explore the optimal dosage for maximizing efficacy and safety.
What evidence suggests that eteplirsen might be an effective treatment for DMD?
Research has shown that eteplirsen can greatly increase the production of dystrophin, a protein that helps muscles function properly, in boys with Duchenne muscular dystrophy (DMD). One study found that patients' dystrophin levels increased to 23% of normal after 24 weeks. In this trial, different doses are being tested: participants in Part 1 will receive 100 mg/kg and 200 mg/kg doses, while Part 2 includes a comparison of 30 mg/kg, 100 mg/kg, and 200 mg/kg doses. The usual dose of 30 mg/kg has demonstrated positive effects over a longer period, suggesting that higher doses might be even more beneficial. Overall, eteplirsen shows promise in helping DMD patients by boosting dystrophin production.26789
Who Is on the Research Team?
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Are You a Good Fit for This Trial?
This trial is for boys with Duchenne Muscular Dystrophy who can walk independently and perform a timed test quickly. They must have specific genetic mutations treatable by skipping exon 51, stable breathing function, and be on steady corticosteroid doses. Boys using other DMD treatments or with kidney issues, recent major surgery, heart problems, or other serious diseases cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by 200 mg/kg once weekly for at least 4 weeks to evaluate safety and tolerability
Dose Finding and Comparison
Participants receive either 30 mg/kg, 100 mg/kg, or 200 mg/kg of eteplirsen once weekly to evaluate efficacy and safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Eteplirsen
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor