Comparing a new treatment with standard care for Duchenne Muscular Dystrophy
A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
This study is testing a new treatment for Duchenne Muscular Dystrophy to see if it works better than the usual glucocorticoid therapy for both walking and non-walking patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Ages | 4 Years and up |
| Sex | Male |
| Sponsor | Sarepta Therapeutics, Inc. Industry-sponsored |
| Locations | 15 sites (Little Rock, Arkansas and 14 other locations) |
| Trial ID | NCT06270719 on ClinicalTrials.gov |
What this trial studies
This observational study collects data on treatment outcomes for Duchenne Muscular Dystrophy (DMD) patients receiving delandistrogene moxeparvovec compared to those on standard glucocorticoid therapy. It is a multicenter study conducted in the United States, focusing on both medical history and prospective data. Participants include both ambulatory and non-ambulatory individuals diagnosed with DMD, who are either starting the new treatment or are on standard care.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 4 years and older with a confirmed diagnosis of DMD who are either starting delandistrogene moxeparvovec or are on chronic glucocorticoid therapy.
Not a fit: Patients with specific genetic deletions in the DMD gene or those currently participating in other interventional studies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide insights into the effectiveness of delandistrogene moxeparvovec compared to traditional treatments for DMD.
How similar studies have performed: Other studies have explored gene therapies for DMD, but this specific observational approach comparing delandistrogene moxeparvovec to standard care is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. A participant recruited to Cohorts 1a or 2: * Is at least 4 years of age at the time of enrollment. * Is ambulatory per protocol specified criteria. A participant recruited to Cohort 1b: - Is non-ambulatory per protocol-specified criteria. For Delandistrogene Moxeparvovec-treated Participants: - Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment. For Comparators: - Is unexposed to DMD gene therapy at the time of study enrollment. Exclusion Criteria: * Has any deletion of exon 8 and/or exon 9 in the DMD gene. * Is currently participating in any DMD interventional study at the time of this observational study enrollment. * Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise: * The participant's ability to comply with the protocol-required procedures, * The participant's wellbeing or safety, and/or * The clinical interpretability of the data collected from the participant. Other inclusion/exclusion criteria may apply.
Where this trial is running
Little Rock, Arkansas and 14 other locations
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
- University of Colorado - PPDS — Aurora, Colorado, United States (Recruiting)
- Children's National Medical Center — Washington, District of Columbia, United States (Recruiting)
- Nicklaus Children's Hospital — Miami, Florida, United States (Recruiting)
- Nemours Children's Hospital - Orlando — Orlando, Florida, United States (Recruiting)
- Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- Indiana Clinical and Translational Science Institute — Indianapolis, Indiana, United States (Recruiting)
- University of Kansas Medical Center — Kansas City, Kansas, United States (Recruiting)
- University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
- Penn State Health Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
- Cook Children's Hospital — Fort Worth, Texas, United States (Recruiting)
- Children's Hospital of the King's Daughters — Norfolk, Virginia, United States (Recruiting)
- The Medical College of Wisconsin — Milwaukee, Wisconsin, United States (Recruiting)
Study contacts
- Study coordinator: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
- Email: SareptAlly@sarepta.com
- Phone: 1-888-SAREPTA (1-888-727-3782)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.