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Gene Transfer Therapy for Duchenne Muscular Dystrophy (EMBARK Trial)

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
Is ambulatory and from 4 to under 8 years of age at time of randomization.
Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to week 104
Awards & highlights

EMBARK Trial Summary

This trial will test if gene transfer therapy is safe and effective in boys with Duchenne Muscular Dystrophy. It is a placebo-controlled study, which means that some participants will receive a placebo instead of the treatment. Those in the placebo group will be given the opportunity to receive gene transfer therapy at the beginning of the second year.

Who is the study for?
This trial is for boys aged 4 to under 8 with Duchenne Muscular Dystrophy (DMD), confirmed by genetic testing. They must be able to do motor tests, on a stable dose of corticosteroids, and not have high rAAVrh74 antibody levels or previous gene therapy.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of delandistrogene moxeparvovec (SRP-9001), a gene transfer therapy against a placebo in boys with DMD. It's randomized and double-blind; those initially receiving placebo may get the therapy after one year.See study design
What are the potential side effects?
Potential side effects are not specified but may include immune reactions due to gene transfer, issues from long-term corticosteroid use, or complications related to underlying DMD.

EMBARK Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 4 and 8 years old and can walk on my own.
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I have been officially diagnosed with Duchenne Muscular Dystrophy.
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I can participate in tests that measure my muscle movements.
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I am between 4 and 7 years old and can walk on my own.
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I have a confirmed diagnosis of Duchenne Muscular Dystrophy.

EMBARK Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 104
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to week 104 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 NSAA Total Score at Week 52
Secondary outcome measures
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device
Part 1: Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 Meter Walk/Run, and the Timed Stair Ascend 4 Steps Test at Week 52
+3 more

EMBARK Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Delandistrogene Moxeparvovec followed by PlaceboExperimental Treatment2 Interventions
Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
Group II: Placebo followed by Delandistrogene MoxeparvovecPlacebo Group2 Interventions
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
placebo
2010
Completed Phase 4
~6580
delandistrogene moxeparvovec
2018
Completed Phase 2
~50

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheIndustry Sponsor
2,428 Previous Clinical Trials
1,088,928 Total Patients Enrolled
Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,413 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,777 Previous Clinical Trials
8,063,360 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05096221 — Phase 3
Duchenne Muscular Dystrophy Research Study Groups: Placebo followed by Delandistrogene Moxeparvovec, Delandistrogene Moxeparvovec followed by Placebo
Duchenne Muscular Dystrophy Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05096221 — Phase 3
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05096221 — Phase 3

Frequently Asked Questions

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

Does this research include patients who are octogenarians or younger?

"The age limit for this study is 7 years old. Any patients who are older than 4 years old can be enrolled."

Answered by AI

How many total test sites are there for this experiment?

"In total, 24 hospitals are running this trial. Some notable locations include UC San Diego Altman Clinical and Translational Research Institute in La Jolla, University of Florida in Gainesville, and University of Utah Hospital in Salt Lake City."

Answered by AI

Does SRP-9001 have any dangerous side effects?

"There is a fair amount of clinical data supporting the safety of SRP-9001, as this is a Phase 3 trial."

Answered by AI

Are patients currently welcome to sign up for this clinical trial?

"According to the latest update on clinicaltrials.gov, this particular trial is not currently recruiting patients. The trial was initially posted on October 27th, 2021 but has not been updated since September 20th, 2022. Although this study is no longer enrolling, there are 88 other trials that are still looking for participants."

Answered by AI

To whom is this research opportunity available?

"This particular medical trial is testing a new medication for people with muscular dystrophies. The requirements for enrollment are as follows: The patient must be ambulatory and between the ages of 4 and 8, have a definitive diagnosis of DMD, the ability to cooperate with motor assessment testing, a stable daily dose of oral corticosteroids, and no elevated rAAVrh74 antibody levels. In addition, the candidate's genetic testing must show a frameshift mutation or premature stop codon contained between exons 18 and 79 (with the exception of mutation fully contained within exon 45)."

Answered by AI

Who else is applying?

How old are they?
18 - 65
< 18
What site did they apply to?
University of Rochester
Boston Children's Hopsital
What portion of applicants met pre-screening criteria?
Met criteria
What state do they live in?
Puerto Rico
Recent research and studies
clinicaltrials.govStudy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
2021. "A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05096221.
All > Mens Health > Muscular Dystrophy
~37 spots leftby Apr 2025
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