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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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current enrollment threshold for this medical experiment?

"This investigation is not accepting any new patients at this time. It was listed on clinicaltrials.gov on the 18th of February 2020 and its most recent update came through on November 28th 2022. If you are searching for alternative trials, there are 86 studies concerning muscular dystrophies that are currently recruiting participants and 4 research projects related to Eteplirsen which have open enrollment periods."

Answered by AI

Are there opportunities at this time for individuals to participate in the trial?

"Unfortunately, the current trial in question is no longer accepting patients. This was initially posted on February 18th 2020 and last edited November 28th 2022. Those searching for similar studies may be interested to learn that there are 86 muscular dystrophy trials recruiting participants as well as 4 open clinical trials involving Eteplirsen."

Answered by AI

Is this the inaugural attempt to investigate such a phenomenon?

"Currently, there are 4 concurrent clinical studies centered around Eteplirsen in 28 different countries and 47 cities. This research was first initiated by Sarepta Therapeutics, Inc.'s phase 3 study with 222 participants back in 2016; since then 10 trials have been concluded."

Answered by AI

Are there additional investigations exploring the effectiveness of Eteplirsen?

"Currently, there are 4 clinical studies in progress for Eteplirsen with 3 of them being Phase 3 trials. Although the majority of these tests are located in Milano and Mazowieckie, overall 157 medical institutions across the world have opened up their doors to this research."

Answered by AI

Are there any potential adverse effects associated with taking Eteplirsen?

"Due to Eteplirsen's Phase 2 status, which indicates the presence of some safety data yet a lack of efficacy information, our team at Power has assigned this drug a score of 2."

Answered by AI
~1 spots leftby Apr 2025