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 nmDMD patients.
Objectives: We aimed to describe the demographics of the STRIDE population and interim safety results, as of January 31, 2022, the latest data cut-off date.
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, 2022, 306 patients had been enrolled in STRIDE in 14 countries and received at least one ataluren dose. Total mean (standard deviation [SD]) exposure to ataluren was 1623 (586.3) days; equivalent to 1359.8 patient-years. Safety outcomes were consistent with the known safety profile of ataluren. Forty-three of the 306 boys discontinued the study. Of the 306 boys enrolled, 290 had genetically confirmed nmDMD. Most patients were white (213/290 [73.4%]) and the mean (SD) age at consent date was 10.2 (4.2) years (n=290). Mean (SD) age at first symptoms was 2.8 (1.7) years (n=273); at nmDMD confirmation it was 5.1 (3.2) years (n=281). Median time between first symptoms and nmDMD confirmation was 1.4 years (n=269). Most patients used concomitant corticosteroids (261/290 [90.0%]).
Conclusions: STRIDE is the first drug registry for nmDMD patients. The interim registry data suggest ataluren has a favorable safety profile when used in routine clinical practice, consistent with that shown in clinical trials.