160 Participants Needed

High-Dose Eteplirsen for DMD

(MIS51ON Trial)

Recruiting at 58 trial locations
MI
ST
Overseen BySarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Age: < 18
Sex: Male
Trial Phase: Phase 3
Sponsor: Sarepta Therapeutics, Inc.
Must be taking: Corticosteroids
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human 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?

Eteplirsen has been generally well tolerated in clinical trials for Duchenne muscular dystrophy, with studies showing it is safe for use in young boys as well.12345

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

MD

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

8 weeks

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

144 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Eteplirsen
Trial Overview The 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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Sarepta Therapeutics, Inc.

Lead Sponsor

Trials
54
Recruited
34,000+

Findings from Research

Eteplirsen (Exondys 51) has received accelerated FDA approval as a treatment for Duchenne muscular dystrophy (DMD) by promoting dystrophin production through exon skipping, specifically targeting exon 51, which is relevant for about 14% of DMD patients.
The review highlights the pharmacological, efficacy, safety, and tolerability data from preclinical and clinical trials, while also addressing concerns regarding the drug's efficacy and its role as a potential therapeutic strategy for DMD.
Eteplirsen in the treatment of Duchenne muscular dystrophy.Lim, KR., Maruyama, R., Yokota, T.[2022]
Eteplirsen significantly increased dystrophin production in boys with Duchenne muscular dystrophy (DMD) after 24 weeks of treatment, with 23% of normal dystrophin levels observed in the 30 mg/kg group, and even higher levels (52% and 43%) at 48 weeks for the 30 and 50 mg/kg groups, respectively.
The treatment also improved walking distance on the 6-minute walk test (6MWT) by an average of 67.3 meters compared to placebo, indicating that longer treatment duration is more important for efficacy than the dosage, and no severe adverse events were reported.
Eteplirsen for the treatment of Duchenne muscular dystrophy.Mendell, JR., Rodino-Klapac, LR., Sahenk, Z., et al.[2022]

References

Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping. [2023]
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. [2022]
Eteplirsen in the treatment of Duchenne muscular dystrophy. [2022]
Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy. [2022]
Eteplirsen: First Global Approval. [2018]
Eteplirsen for the treatment of Duchenne muscular dystrophy. [2022]
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