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Gene Therapy for Duchenne Muscular Dystrophy (ENVISION Trial)
Phase 3
Recruiting
Research Sponsored by Sarepta Therapeutics, Inc.
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 baseline up to week 124
Awards & highlights
ENVISION Trial Summary
This trial studies a gene therapy to treat Duchenne Muscular Dystrophy in boys. It's a 2-part, randomized, double-blind, placebo-controlled study lasting 128 weeks. Participants may get IV SRP-9001.
Who is the study for?
This trial is for non-ambulatory and ambulatory males with Duchenne Muscular Dystrophy (DMD), aged ≥8 to <18, who can perform motor assessment tests. They must be on a stable dose of corticosteroids and have specific genetic mutations in DMD. Those with high antibodies against the therapy vector or prior gene therapy are excluded.Check my eligibility
What is being tested?
The study tests delandistrogene moxeparvovec, a gene transfer therapy for DMD. Participants will either receive this treatment or a placebo via IV over approximately 128 weeks in a randomized, double-blind setup where neither they nor the researchers know who gets what until after the study.See study design
What are the potential side effects?
While not specified here, gene therapies like delandistrogene moxeparvovec may cause immune reactions, potential liver issues, and site-specific reactions at infusion points among other risks that would be monitored throughout the trial.
ENVISION Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline up to week 124
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 124
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 the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Secondary outcome measures
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
+4 moreENVISION Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Delandistrogene Moxeparvovec followed by PlaceboExperimental Treatment2 Interventions
Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.
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 approximately 72 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
delandistrogene moxeparvovec
2018
Completed Phase 2
~50
placebo
2010
Completed Phase 4
~6580
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Who is running the clinical trial?
Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,391 Total Patients Enrolled
Hoffmann-La RocheIndustry Sponsor
2,435 Previous Clinical Trials
1,092,250 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,793 Previous Clinical Trials
8,068,466 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I haven't had gene therapy or experimental drugs to boost dystrophin recently.I am between 8 and 18 years old and can walk on my own.I am between 8 and 18 years old and can walk on my own.I cannot walk by myself according to the study's specific rules.My genetic test shows a specific mutation in my DNA.My body does not have high levels of antibodies against rAAVrh74.I have been on a stable dose of oral corticosteroids for the last 12 weeks.I have a confirmed diagnosis of Duchenne Muscular Dystrophy.I can participate in tests that measure my muscle movements.Your blood doesn't have high levels of antibodies against a specific virus called rAAVrh74, as outlined in the study guidelines.I have been on a stable dose of oral corticosteroids for at least 12 weeks.My genetic test shows a specific mutation in my DNA.I cannot walk by myself according to the study's specific rules.
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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