What is the clinical evidence from this study?

Study design: Other. Population: Duchenne muscular dystrophy (n=218). Intervention: Delandistrogene moxeparvovec vs. Placebo. Primary outcome: Cardiac safety (adverse events, echocardiography, troponin I).

What question did this study set out to answer?

To assess the cardiac safety of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy over five years.

July 3, 2026Open Access

Cardiac Safety Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy with Up to 5 Years of Follow-up

Resultado clave

Delandistrogene moxeparvovec showed a manageable cardiac safety profile in 216 treated patients with DMD over up to 5 years, with 2 cases of resolving myocarditis and no persistent cardiac injury.

Puntos clave

To assess the cardiac safety of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy over five years.
Data collected from four clinical trials (NCT03375164, NCT03769116, NCT04626674, NCT05096221) with up to 5 years follow-up.
Adverse events, cardiac echocardiography, and troponin I were assessed regularly.
Patients with left ventricular ejection fraction < 40% were excluded.
Of 218 patients, only 2 cases of myocarditis were reported, both resolving within 3 weeks.
96% of the cohort were ambulatory, with the majority maintaining stable LVEF.
Cardiac MRI showed no significant changes in heart function between treatment and placebo at 1 and 2 years.

PICO estructurado

Does delandistrogene moxeparvovec have a manageable cardiac safety profile in patients with Duchenne muscular dystrophy?

Población

218 predominantly ambulatory patients (mean age 6.4 years) with Duchenne muscular dystrophy and LVEF ≥ 40%, followed for up to 5 years to assess cardiac safety.

Intervención

Delandistrogene moxeparvovec infusion

Comparador

Placebo infusion (in EMBARK substudy) or no comparator

Resultado

Cardiac safety (adverse events, echocardiography, troponin I, cardiac MRI)safety

Delandistrogene moxeparvovec demonstrates a manageable cardiac safety profile with no signs of persistent treatment-related cardiac injury in predominantly younger, ambulatory patients with DMD over up to 5 years of follow-up.

Limitaciones

Subclinical fibrosis cannot be ruled out

Resumen

INTRODUCTION: In patients with Duchenne muscular dystrophy (DMD), the gene transfer therapy delandistrogene moxeparvovec delivers a functional form of dystrophin, which has been shown to stabilize or slow disease progression. METHODS: We assessed cardiac safety of delandistrogene moxeparvovec in clinical trials with ≤ 5 years of follow-up. Data were collected from clinical trials 101 (NCT03375164, n = 4), 102 (NCT03769116, n = 41), ENDEAVOR (NCT04626674, n = 48), and EMBARK (NCT05096221, n = 125), which excluded patients with left ventricular ejection fraction (LVEF) 50%. Although cardiac MRI without gadolinium revealed no relevant differences in heart function between patients 1 or 2 years after delandistrogene moxeparvovec versus patients 1 year after placebo infusion, subclinical fibrosis cannot be ruled out. CONCLUSION: Results from delandistrogene moxeparvovec trials with 1 to 5 years of follow-up suggest a manageable cardiac safety profile in this study population of predominantly younger, ambulatory patients with DMD who had no signs of persistent treatment-related cardiac injury.

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