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
M
Marcia Felker, MD
Primary Investigator
Enrolling By Invitation
4 years - 5 years
Male
Phase
4
2 participants needed
1 Location
Brief description of study
What is the purpose of this study?
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice. In addition, treatment outcomes will be collected prospectively from post-trial participants who have received delandistrogene moxeparvovec through participation in select SRP-9001 studies.
THIS STUDY IS ENROLLING BY INVITATION ONLY - DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.
Detailed description of study
What will happen during the study?
Participants were either (1) prescribed delandistrogene moxeparvovec commercially as part of clinical care prior to entry into this trial or (2) previously received delandistrogene moxeparvovec through participation in select SRP-9001 studies.
Participants unexposed to DMD gene therapy receiving standard of care therapy (chronic glucocorticoid treatment) that does not include delandistrogene moxeparvovec or other DMD gene therapies.
Participants unexposed to DMD gene therapy receiving standard of care therapy (chronic glucocorticoid treatment) that does not include delandistrogene moxeparvovec or other DMD gene therapies.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Duchenne Muscular Dystrophy, Riley
-
Age: 4 years - 5 years
-
Gender: Male
Inclusion Criteria:
1. Is male at birth
2. Among participants recruited from routine clinical practice: Is aged 4 through 5 years at
the time of enrollment
Note: There is no age restriction for post-trial participants.
3. Capable of giving signed informed consent/assent (if applicable), which includes
compliance with the requirements and restrictions listed in the consent form and in this
protocol and has a parent(s) or legal guardian(s) who is (are) willing to provide informed
consent for the participant to participate in the study and comply with study data collection
procedures.
4. Has an established clinical diagnosis of DMD based on documentation of clinical findings
and prior confirmatory genetic testing using a clinical diagnostic genetic test.
5. Among participants recruited from routine clinical practice: Is ambulant (defined as being
able to complete the 10MWR in less than 30 seconds) at screening
Note: There is no ambulation restriction for post-trial participants.
6. Among participants recruited from routine clinical practice: Is currently receiving or has
been prescribed to start chronic glucocorticoid therapy at the time of this observational
study enrollment
Note: There is no glucocorticoid restriction for post-trial participants.
1. Is male at birth
2. Among participants recruited from routine clinical practice: Is aged 4 through 5 years at
the time of enrollment
Note: There is no age restriction for post-trial participants.
3. Capable of giving signed informed consent/assent (if applicable), which includes
compliance with the requirements and restrictions listed in the consent form and in this
protocol and has a parent(s) or legal guardian(s) who is (are) willing to provide informed
consent for the participant to participate in the study and comply with study data collection
procedures.
4. Has an established clinical diagnosis of DMD based on documentation of clinical findings
and prior confirmatory genetic testing using a clinical diagnostic genetic test.
5. Among participants recruited from routine clinical practice: Is ambulant (defined as being
able to complete the 10MWR in less than 30 seconds) at screening
Note: There is no ambulation restriction for post-trial participants.
6. Among participants recruited from routine clinical practice: Is currently receiving or has
been prescribed to start chronic glucocorticoid therapy at the time of this observational
study enrollment
Note: There is no glucocorticoid restriction for post-trial participants.
Updated on
28 Apr 2025.
Study ID: NEUR-SAREPTA-SRP-9001-401, 21287
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