Casimersen for Duchenne Muscular Dystrophy

Phase 3

Waitlist Available

Research Sponsored by Sarepta Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 30 days after the last infusion of study drug (assessed up to 148 weeks)

Awards & highlights

Study Summary

This trial is testing if two drugs are safe and tolerated by patients with Duchenne muscular dystrophy when taken long-term.

Eligible Conditions

Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 30 days after the last infusion of study drug (assessed up to 148 weeks)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 30 days after the last infusion of study drug (assessed up to 148 weeks)

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 30 days after the last infusion of study drug (assessed up to 148 weeks) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Patients With Serious Adverse Events (SAEs)

Trial Design

2Treatment groups

Experimental Treatment

Group I: GolodirsenExperimental Treatment1 Intervention

Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.

Group II: CasimersenExperimental Treatment1 Intervention

Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.

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Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor

49 Previous Clinical Trials

33,368 Total Patients Enrolled

Medical DirectorStudy DirectorSarepta Therapeutics, Inc.

2,788 Previous Clinical Trials

8,065,881 Total Patients Enrolled

~25 spots leftby Jun 2025

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