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Antisense Oligonucleotide

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

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
No Placebo-Only Group

Summary

This trial tests AMONDYS 45, EXONDYS 51, and VYONDYS 53 in boys with Duchenne muscular dystrophy who have specific genetic mutations. The medications aim to correct these mutations by skipping duplicated parts of the gene to produce a normal muscle protein. The study will evaluate safety and effectiveness over several months.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
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 study objectives
Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen).

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
52 Previous Clinical Trials
33,965 Total Patients Enrolled
Kevin Flanigan, MDPrincipal InvestigatorNationwide Children's Hospital
4 Previous Clinical Trials
29 Total Patients Enrolled
~1 spots leftby Dec 2025
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