Background: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by a lack of functional dystrophin. Ataluren promotes readthrough of an in-frame premature stop codon to produce full-length dystrophin and is indicated for the 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.
Objective: We aimed to describe the demographics of the STRIDE population and the interim safety results, as of January 31, 2021.
Methods: Data from enrolled patients are collected at the consent date; for patients who initiated ataluren as part of a commercial or early access program before enrollment, data for the period prior to 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 in STRIDE in 13 countries and received at least one ataluren dose. Total mean (standard deviation [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. Of the 286 boys enrolled, 269 had genetically confirmed nmDMD. Most patients were Caucasian (194/269 [72.1%]) and the mean (SD) age at consent date 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%]).
Conclusions: STRIDE is the first drug registry for nmDMD patients. The interim registry data suggest that ataluren has a favorable safety profile when used in routine clinical practice, which is consistent with that shown in clinical trials.