DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results.

ABSTRACT:

Background

Pharmacokinetic/pharmacodynamic modeling indicates that the higher dose of nusinersen may be associated with a clinically meaningful increase in efficacy above that seen with the 12-mg approved dose.

Objective

Here we describe both the design of DEVOTE (NCT04089566), a 3-part clinical study evaluating safety, tolerability, and efficacy of higher dose of nusinersen, and results from the initial Part A.

Methods

DEVOTE Part A evaluates safety and tolerability of a higher nusinersen dose; Part B assesses efficacy in a randomized, double-blind design; and Part C assesses safety and tolerability of participants transitioning from the 12-mg dose to higher doses.

Results

In the completed Part A of DEVOTE, all 6 enrolled participants aged 6.1-12.6 years have

SUBMITTER: Finkel RS 

PROVIDER: S-EPMC10578235 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature