An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

Conditions

• Duchenne Muscular Dystrophy

Eligibility Criteria

Sex

MALE

Age

4 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Delandistrogene Moxeparvovec — GENETIC
  No study medication will be provided by the sponsor during this study.
• Standard of Care — DRUG
  No study medication will be provided by the sponsor during this study.

Study Details

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.

Key Dates

Start date

Feb 7, 2024

Status verified

Apr 2026

Primary completion

Dec 31, 2029

Completion

Dec 31, 2038

Study Design

Enrollment

500 participants (estimated)

Arms

• Arm: Cohort 1 (Treated)
  Cohort 1: participants prescribed ELEVIDYS in a commercial setting. Cohort 1a (Ambulatory ELEVIDYS Prospectively Treated Cohort): participants prescribed ELEVIDYS by commercially treating physicians with consent no later than 30 days from infusion. All participants will be dosed with ELEVIDYS based on United States prescribing information (USPI). The Post Market Requirement (PMR) Cohort (sub-cohort of 1a) consists of participants with laboratory data as specified in the ELEVIDYS USPI. Cohort 1b (Non-ambulatory ELEVIDYS Prospectively Treated Cohort): non-ambulatory DMD participants prescribed ELEVIDYS commercially and recruited by treating physicians before infusion (enrollment currently closed). Cohort 1c (ELEVIDYS Retrospectively Treated Cohort): participants dosed with ELEVIDYS with complete baseline data within 6 months prior to dosing and complete prospectively collected annual follow-up data after infusion until the time of cohort entry (sponsor approval required for enrollment).
• Arm: Cohort 2 (Standard of Care)
  Cohort 2: ambulatory DMD participants who are at least 4 years of age at baseline, unexposed to DMD gene therapy, and receiving or prescribed chronic glucocorticoids at study entry.

Primary Outcome Measure

Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 [ Time Frame: Baseline, Month 12 ]

Locations (25)

Find similar trials in Little Rock, AR

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