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Gene Therapy for Duchenne Muscular Dystrophy (ENVISION Trial)
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.
ENVISION Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.ENVISION Trial Design
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Who is running the clinical trial?
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- 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.
- Group 1: Placebo followed by Delandistrogene Moxeparvovec
- Group 2: Delandistrogene Moxeparvovec followed by Placebo
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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.
Frequently Asked Questions
What can be said about the safety profile of this remedy?
"Our assessment of the safety profile for this medication is a 3, as it has been tested through Phase 3 trials. This suggests that there are multiple levels of evidence to back up its efficacy and security."
In how many distinct venues is this clinical trial being administered?
"This medical trial is currently recruiting subjects from 14 different sites, such as Gainesville, Baltimore and Little Rock. To alleviate the burden of travel, it is important to choose a clinic near your residence if you plan on participating."
Are there any vacancies left in this clinical experiment?
"The records on clinicaltrials.gov state that this medical trial, introduced to the portal on June 30th 2023, is not accepting new patients at this time. However, there are 93 other trials actively recruiting participants currently."
Who else is applying?
What site did they apply to?
What portion of applicants met pre-screening criteria?
Why did patients apply to this trial?
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