Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.

ABSTRACT:

Objective

This double-blind, randomized, placebo-controlled Phase 2 study (NCT01462292) assessed the 24-week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24-week off-dose persistent effect, in ambulant Duchenne muscular dystrophy (DMD) patients.

Methods

Male DMD patients (≥5 years; time to rise from floor ≤15 s) were randomized to drisapersen 3 mg/kg/week, 6 mg/kg/week or placebo. The primary efficacy endpoint was change from baseline in 6-minute walking distance (6MWD) at week 24. Secondary endpoints included changes in timed function tests, muscle strength, and pulmonary function tests.

Results

Fifty-one patients were randomized to placebo (N = 16), drisapersen 3 mg/kg/week (N = 17) or 6

SUBMITTER: McDonald CM 

PROVIDER: S-EPMC6093847 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature