Results of a randomized double-blind study evaluating luvadaxistat in adults with Friedreich ataxia.

ABSTRACT:

Objectives

Friedreich ataxia (FRDA) is a rare disorder with progressive neurodegeneration and cardiomyopathy. Luvadaxistat (also known as TAK-831; NBI-1065844), an inhibitor of the enzyme d-amino acid oxidase, has demonstrated beneficial effects in preclinical models relevant to FRDA. This phase 2, randomized, double-blind, placebo-controlled, parallel-arm study evaluated the efficacy and safety of oral luvadaxistat in adults with FRDA.

Methods

Adult patients with FRDA were randomized 2:1:2 to placebo, luvadaxistat 75 mg twice daily (BID), or luvadaxistat 300 mg BID for 12 weeks. The primary endpoint changed from baseline at week 12 on the inverse of the time to complete the nine-hole peg test (9-HPT^-1 ), a performance-based measure of the function of the upper ex

SUBMITTER: Wang H 

PROVIDER: S-EPMC8164851 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature