delandistrogene moxeparvovec
GeneticNo study medication will be provided by the sponsor during this study.
Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
NCT Number: NCT06270719
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec (ELEVIDYS) as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at the time of study enrollment in routine clinical practice.
Interested in participating?
Request Info4 year and older
Male
Observational
Arkansas Children's Hospital, Little Rock, Arkansas, United States
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
For Standard of Care Comparators (Cohort 2):
Exclusion criteria
Other inclusion/exclusion criteria may apply.
No study medication will be provided by the sponsor during this study.
Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
No study medication will be provided by the sponsor during this study.
Other names: Chronic glucocorticoid treatment
Time frame: Baseline, Month 12
Time frame: Baseline through Month 12
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Baseline through Month 12
Time frame: Up to 10 years
Time frame: Up to 10 years
Time frame: Up to 10 years
Sarepta Therapeutics, Inc.
Industry
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
Acronym: ENDURE
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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