Delandistrogene Moxeparvovec for Duchenne Muscular Dystrophy
(ENDURE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to understand how a new treatment, delandistrogene moxeparvovec (a gene therapy), compares to the usual medications for Duchenne muscular dystrophy (DMD), a genetic disorder that causes muscle weakness. Participants will either receive this new gene therapy or continue with standard treatment, including medications like glucocorticoids. This study is suitable for those diagnosed with DMD, currently on or starting glucocorticoid treatment, and at least 4 years old. The trial seeks to gather insights on how these treatments affect patients' health and daily lives. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.
Do I need to stop my current medications for the trial?
The trial does not specify if you need to stop your current medications. However, it mentions that participants should be on or starting chronic glucocorticoid therapy, so you may need to continue that medication.
What is the safety track record for delandistrogene moxeparvovec and chronic glucocorticoid treatment?
Research has shown that delandistrogene moxeparvovec is generally safe for patients with Duchenne muscular dystrophy (DMD). In earlier studies, this gene therapy demonstrated a good safety record. Most patients did not experience serious side effects, and any issues were manageable. For example, real-world data from 11 young patients who received this treatment reported no severe problems. This suggests the treatment is safe, with ongoing monitoring to ensure optimal results.12345
Why are researchers enthusiastic about this study treatment?
Delandistrogene moxeparvovec is unique because it offers a gene therapy approach to treating Duchenne Muscular Dystrophy (DMD), unlike the standard of care, which primarily involves chronic glucocorticoid treatment. This investigational treatment works by delivering a functional copy of the dystrophin gene, aiming to address the root cause of DMD rather than just managing symptoms. Researchers are excited about this treatment because it has the potential to significantly improve muscle function and slow disease progression, offering hope for a more effective, long-term solution for those affected by DMD.
What evidence suggests that this trial's treatments could be effective for Duchenne muscular dystrophy?
Research has shown that delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), may help maintain or improve movement abilities. In this trial, participants in Cohort 1 will receive delandistrogene moxeparvovec. Studies found that after one year, patients who received this treatment had stable or improved movement. Longer-term results, up to five years, suggest this therapy can significantly slow the progression of DMD symptoms. The treatment delivers a small gene to muscle cells, which helps maintain muscle function over time. Overall, the evidence is encouraging for those considering this treatment option.46789
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observation
Participants' medical history and treatment outcomes are collected and observed
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Delandistrogene Moxeparvovec
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Cohort 2 includes ambulatory DMD participants aged 4 years or greater at baseline unexposed to DMD gene therapy and receiving standard of care therapy (chronic glucocorticoid treatment) at study entry with or without baseline use of other DMD approved therapies.
Participants in Cohort 1 will have a plan for prescription of delandistrogene moxeparvovec commercially as part of clinical care prior to entry into this study. Cohort 1a includes participants who are ambulatory and aged 4 years or greater at baseline prescribed delandistrogene moxeparvovec commercially and recruited by treating physicians before infusion is administered. Cohort 1b includes non-ambulatory DMD participants aged 4 years or greater at baseline prescribed delandistrogene moxeparvovec commercially and recruited by treating physicians before infusion is administered.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor
Citations
Delandistrogene Moxeparvovec Gene Therapy in ...
This Evidence in Focus reviews the current evidence on the efficacy and adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular ...
1-Year Interim Results from Study SRP-9001-103 ...
One-year post-treatment, delandistrogene moxeparvovec was well tolerated, and demonstrated stabilized or improved motor function, suggesting a clinical benefit ...
3.
investorrelations.sarepta.com
investorrelations.sarepta.com/static-files/a479a06b-7f40-495b-b540-96fde84f6725Five-year outcomes with delandistrogene moxeparvovec in ...
Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD):. A Phase 1/2a study. JR Mendell,1,2 ...
4.
ajmc.com
ajmc.com/view/delandistrogene-moxeparvovec-shows-long-term-impacts-at-3-and-5-years-in-dmdDelandistrogene Moxeparvovec Shows Long-Term ...
Delandistrogene moxeparvovec showed significant long-term stabilization or slowed progression in DMD patients over 3 and 5 years compared to ...
AAV gene therapy for Duchenne muscular dystrophy
In these studies, delandistrogene moxeparvovec micro-dystrophin expression was robust with sarcolemmal localization up to 60 weeks after ...
6.
investorrelations.sarepta.com
investorrelations.sarepta.com/static-files/a9bf712a-d35f-465c-a86f-466b15f1b314Five-Year Outcomes With Delandistrogene Moxeparvovec in ...
to evaluate the safety of delandistrogene moxeparvovec ... Analysis Project; DMD, Duchenne muscular dystrophy; EC, external control; FOR-.
A phase 1/2a nonrandomized trial
Gene transfer therapy with delandistrogene moxeparvovec treatment is well tolerated, with a favorable safety profile. Functional improvements are sustained ...
Real-world outcomes of delandistrogene moxeparvovec ...
Herein, we report real-world data of 11 ambulatory patients with DMD, ages 4–6, treated with commercial delandistrogene moxeparvovec. Patients ...
NCT06270719 | An Observational Study Comparing ...
The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving ...
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