clinical trial · NCT06270719
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular DystrophyDelandistrogene MoxeparvovecStandard of Care
brief summary
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.
started
Feb 7, 2024
primary completion
Dec 31, 2029
completion
Dec 31, 2038
last updated
May 4, 2026
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol