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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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline up to week 104
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 104
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 moreEMBARK 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
2,434 Previous Clinical Trials
1,091,266 Total Patients Enrolled
Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,413 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,789 Previous Clinical Trials
8,067,204 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been officially diagnosed with Duchenne Muscular Dystrophy.I can participate in tests that measure my muscle movements.I am between 4 and 7 years old and can walk on my own.Your medical tests show something abnormal according to the study's rules.I have a confirmed diagnosis of Duchenne Muscular Dystrophy.Your antibody levels are not too high according to the study's rules.I have been on a stable dose of oral corticosteroids for at least 12 weeks.I am between 4 and 8 years old and can walk on my own.I can participate in tests that measure my muscle movements.I have been on a stable dose of oral corticosteroids for at least 12 weeks.I have not used any experimental drugs or treatments to increase dystrophin recently.Your blood test shows that you don't have high levels of rAAVrh74 antibodies as required by the study.My genetic test shows a specific mutation in my DNA, not including exon 45.My genetic test shows a specific mutation in my DNA, not including exon 45.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo followed by Delandistrogene Moxeparvovec
- Group 2: Delandistrogene Moxeparvovec followed by Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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