Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE Registry

Objectives: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by a lack of functional dystrophin. Ataluren is indicated for treatment of patients with nonsense mutation (nm) DMD. Strategic Targeting of Registries and International Database of Excellence (STRIDE; NCT02369731) is an ongoing registry providing real-world data on ataluren use in patients with nmDMD. Here, we describe STRIDE population demographics and interim safety results, as of the data cut-off of January 31, 2021.

Methods: Data from enrolled patients are collected at the consent date; data for patients who initiated ataluren before enrollment are obtained retrospectively. Patients will be followed up for ≥5 years or until study withdrawal.

Results: As of January 31, 2021, 286 boys had been enrolled and received at least one ataluren dose. Total mean (SD) ataluren exposure was 1352 (517) days; equivalent to 1059 patient-years. Safety outcomes were consistent with the known safety profile of ataluren. Thirty-one of the 286 boys discontinued the study. nmDMD was genetically confirmed in 269/286 boys. Most patients were Caucasian (194/269 [72.1%]); mean (SD) age at consent was 9.9 (3.8) years (n=269). Mean (SD) age at first symptoms was 2.7 (1.7) years (n=244); at nmDMD confirmation, it was 4.9 (2.7) years (n=260). Median time between first symptoms and nmDMD confirmation was 1.4 years (n=240). Most patients used concomitant corticosteroids (237/269 [88.1%]).

Conclusion: STRIDE is the first drug registry for nmDMD patients. Interim data suggest that ataluren has a favorable safety profile in routine clinical practice, consistent with that shown in clinical trials.