Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.

ABSTRACT:

Objectives

This ongoing, open-label extension study is evaluating the long-term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD).

Methods

The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS]), receiving givinostat or placebo, or were successfully screened but not randomized into EPIDYS. All receive givinostat oral suspension open-label at a flexible, weight-based dose in addition to systemic corticosteroids, and attend visits every 4 months.

Results

A total of 194 patients are included in the current analyses, with a mean duration of givinostat exposure (excluding use in prior studies) of 559.6 days (SD 373.0); when in

SUBMITTER: McDonald CM 

PROVIDER: S-EPMC12623843 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature