High-Dose Eteplirsen for DMD
(MIS51ON Trial)

Recruiting in Palo Alto (17 mi)

+58 other locations

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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

I am between 4 to 6 years old and do not need a ventilator.

I have been on a consistent dose of oral corticosteroids for the last 12 weeks.

I can walk and complete a specific physical task in 10 seconds or less.

See 10 more

Exclusion Criteria

I have kidney problems or other serious health issues.

My heart's pumping ability is reduced, or my heart rhythm test shows a delay.

I have not used experimental drugs for DMD, except certain ones recently.

See 3 more

Treatment Details

Interventions

Eteplirsen (Exon Skipping Agent)

Trial OverviewThe study tests two high doses of Eteplirsen (100 mg/kg and 200 mg/kg) against a standard dose (30 mg/kg) in boys with Duchenne Muscular Dystrophy. It's divided into two parts: first to see if the high doses are safe and then to find out which one works best compared to the standard dose.

Participant Groups

4Treatment groups

Experimental Treatment

Active Control

Group I: Part 2: Eteplirsen 200 mg/kgExperimental Treatment1 Intervention

Randomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks.

Group II: Part 2: Eteplirsen 100 mg/kgExperimental Treatment1 Intervention

Randomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks.

Group III: Part 1: EteplirsenExperimental Treatment1 Intervention

Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.

Group IV: Part 2: Eteplirsen 30 mg/kgActive Control1 Intervention

Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.