High-Dose Eteplirsen for DMD
(MIS51ON Trial)
Trial Summary
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 eteplirsen safe for humans?
How is the drug eteplirsen unique for treating Duchenne muscular dystrophy?
Eteplirsen is unique because it is specifically designed to skip exon 51 in the DMD gene, which helps increase dystrophin production, a protein crucial for muscle function. It is administered intravenously and has been shown to slow down the decline in muscle and lung function in patients with Duchenne muscular dystrophy.12456
What is the purpose of this trial?
This trial is testing eteplirsen, a medication for Duchenne Muscular Dystrophy (DMD). It targets patients with specific genetic mutations that can be treated by skipping exon 51. Eteplirsen helps the body make a protein needed for muscle function by bypassing a faulty part of the gene. Eteplirsen has been approved for treating DMD by skipping exon 51, allowing for the production of a functional dystrophin protein.
Research Team
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Eligibility Criteria
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
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
Treatment Details
Interventions
- Eteplirsen
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor