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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy (ENDEAVOR Trial)

Phase 1
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
Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing
Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 2 up to week 156
Awards & highlights

ENDEAVOR Trial Summary

This trial is testing a new gene therapy for Duchenne Muscular Dystrophy, to see if it is safe and effective.

Who is the study for?
This trial is for boys with Duchenne Muscular Dystrophy (DMD). Different age groups can join: 4-8, ≥8 to <18 years old, and some who don't need steroids yet. They must be able to do motor tests and not have taken gene therapy or certain drugs recently. Those on stable steroid doses can also participate.Check my eligibility
What is being tested?
The study is testing delandistrogene moxeparvovec, a gene transfer therapy for DMD. It's an open-label study where everyone gets the treatment to see if it's safe and how well it works over about three years.See study design
What are the potential side effects?
Possible side effects aren't listed here but typically include immune reactions due to gene transfer, potential liver issues from viral vectors used in the therapy, and general risks associated with genetic therapies.

ENDEAVOR Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been officially diagnosed with Duchenne Muscular Dystrophy.
Select...
I have been officially diagnosed with Duchenne Muscular Dystrophy.

ENDEAVOR Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 2 up to week 156
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 2 up to week 156 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part 1: Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Secondary outcome measures
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
+4 more

ENDEAVOR Trial Design

1Treatment groups
Experimental Treatment
Group I: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
delandistrogene moxeparvovec
2018
Completed Phase 2
~50

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,481 Total Patients Enrolled
Hoffmann-La RocheIndustry Sponsor
2,429 Previous Clinical Trials
1,089,344 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,777 Previous Clinical Trials
8,063,429 Total Patients Enrolled

Media Library

SRP-9001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04626674 — Phase 1
Duchenne Muscular Dystrophy Research Study Groups: Delandistrogene Moxeparvovec
Duchenne Muscular Dystrophy Clinical Trial 2023: SRP-9001 Highlights & Side Effects. Trial Name: NCT04626674 — Phase 1
SRP-9001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04626674 — Phase 1

Frequently Asked Questions

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

Are there a multitude of hospitals conducting this scientific experiment within the state's borders?

"Currently, this trial has 5 available sites, such as Washington University in St. Louis and Stanford University in Palo Alto. Additionally, there are 3 other locations including Children's Hospital of The King's Daughters in Norfolk."

Answered by AI

What are the ramifications of SRP-9001 consumption for human health?

"Since SRP-9001 is currently in Phase 1 of its trial process, there exists only limited evidence demonstrating its efficacy and safety. On a scale from one to three, our experts at Power assigned it with a score of one."

Answered by AI

Are there any vacancies for volunteers in this trial?

"This medical experiment, which was initially posted in November of 2020 and last edited in September 2022, is no longer searching for volunteers. Nonetheless, there are 86 other trials actively enrolling at this time according to clinicaltrials.gov data."

Answered by AI

Who else is applying?

What state do they live in?
California
How old are they?
18 - 65
< 18
What site did they apply to?
Stanford University
University of California, Davis
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

I want my child to have normal life. I had tested one drug so now I want to give it a try.
PatientReceived 1 prior treatment

What questions have other patients asked about this trial?

I m from India. I will travel to the site you told. So how many days I have to stay there?
PatientReceived 1 prior treatment
Recent research and studies
clinicaltrials.govStudy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
2020. "A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04626674.
All > Muscular Dystrophy
~8 spots leftby Nov 2024
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