Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

ABSTRACT:

Importance

Vamorolone is a synthetic steroidal drug with potent anti-inflammatory properties. Initial open-label, multiple ascending dose-finding studies of vamorolone among boys with Duchenne muscular dystrophy (DMD) found significant motor function improvement after 6 months treatment in higher-dose (ie, ≥2.0 mg/kg/d) groups.

Objective

To investigate outcomes after 30 months of open-label vamorolone treatment.

Design, setting, and participants

This nonrandomized controlled trial was conducted by the Cooperative International Neuromuscular Research Group at 11 US and non-US study sites. Participants were 46 boys ages 4.5 to 7.5 years with DMD who completed the 6-month dose-finding study. Data were analyzed from July 2020 through November 2021.

Interventions

Pa

SUBMITTER: Mah JK 

PROVIDER: S-EPMC8790668 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature