SMA – THERAPY: P.265 Escalating dose and randomized, controlled study of high-dose nusinersen in SMA; study design and updated enrollment for the DEVOTE Study

Abstract

The Phase 2/3 DEVOTE study (NCT04089566) is a 3-part study that will examine the safety and efficacy of nusinersen administered intrathecally at higher doses in participants with 5q spinal muscular atrophy (SMA). Part A is an open-label safety evaluation of later onset SMA participants (n=6, age 2-15 years, inclusive; SMA onset age >6 months) who will receive three 28 mg loading doses at 14-day intervals followed by two 28 mg maintenance doses every 4 months. After safety evaluation, Part B will enrol ≤126 participants with infantile-onset (age ≤7 months at informed consent; 2 SMN2 copies; SMA onset age ≤6 months) or later-onset SMA (age 2 to <10 years; SMA onset age >6 months; sat but not walking independently; HFMSE score ≥10 to ≤54).