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Antisense Oligonucleotide

EXONDYS 51 for Duchenne Muscular Dystrophy

Phase 2
Waitlist Available
Led By Kevin Flanigan, MD
Research Sponsored by Kevin Flanigan
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial will test the efficacy and safety of 3 drugs for treating boys with Duchenne Muscular Dystrophy who have a duplication of 1 of 3 exons. There will be weekly infusions and 2 muscle biopsies.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen)
Monitoring for the Development of Unacceptable Toxicity.
Secondary outcome measures
Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen).

Trial Design

3Treatment groups
Experimental Treatment
Group I: VYONDYS 53Experimental Treatment1 Intervention
This arm will involve the treatment of boys with DMD who have a duplication of exon 53, for which VYONDYS 53 will target skipping of this exon.
Group II: EXONDYS 51Experimental Treatment1 Intervention
This arm will involve the treatment of boys with DMD who have a duplication of exon 51, for which EXONDYS 51 will target skipping of this exon.
Group III: AMONDYS 45Experimental Treatment1 Intervention
This arm will involve the treatment of boys with DMD who have a duplication of exon 45, for which AMONDYS 45 will target skipping of this exon.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vyondys 53
2020
Completed Phase 2
~10
Amondys 45
2020
Completed Phase 2
~10
Exondys 51
2020
Completed Phase 2
~10

Find a Location

Who is running the clinical trial?

Kevin FlaniganLead Sponsor
3 Previous Clinical Trials
4 Total Patients Enrolled
Sarepta Therapeutics, Inc.Industry Sponsor
49 Previous Clinical Trials
33,536 Total Patients Enrolled
Kevin Flanigan, MDPrincipal InvestigatorNationwide Children's Hospital
4 Previous Clinical Trials
29 Total Patients Enrolled
~1 spots leftby Jun 2025
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