Casimersen + Golodirsen for Duchenne Muscular Dystrophy

(ESSENCE Trial)

Recruiting in Palo Alto (17 mi)

+75 other locations

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

Research Team

Medical Director

Principal Investigator

Sarepta Therapeutics, Inc.

Eligibility Criteria

This trial is for individuals with Duchenne Muscular Dystrophy (DMD) who have specific genetic deletions treatable by skipping exon 45 or exon 53. Participants must be on a stable dose of corticosteroids, have certain muscle function, and maintain a specific range in the 6-minute walk test. They should not have had gene therapy or other recent experimental treatments.

Inclusion Criteria

I have been on a stable dose of oral corticosteroids for at least 24 weeks.

My lung function is at least half of what is expected for someone my age and size.

I can walk between 300 to 450 meters in 6 minutes.

Exclusion Criteria

I have been treated with SMT C1100 recently and PRO045, PRO053, or PRO051 in the last 6 months.

Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1

I have not had major surgery in the last 3 months.

Treatment Details

Interventions

• Placebo (Drug)
• SRP-4045 (Antisense Oligonucleotide)
• SRP-4053 (Antisense Oligonucleotide)

Trial Overview The study tests the effectiveness of two drugs, SRP-4045 (casimersen) and SRP-4053 (golodirsen), against a placebo in improving DMD symptoms related to specific genetic mutations. The goal is to see if these drugs can help skip over faulty parts of the gene.

Participant Groups

Group I: SRP-4053 Experimental Treatment 1 Intervention

Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Group II: SRP-4045 Experimental Treatment 1 Intervention

Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Group III: Placebo followed by SRP-4045 or SRP-4053 Placebo Group 3 Interventions

Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Who Is Running the Clinical Trial?

Sarepta Therapeutics, Inc.

Lead Sponsor

Trials

54

Recruited

34,000+