Highlights
In this single-centre, 2 × 2 factorial, randomised phase 2 trial, the combination of nicotinamide riboside and individualized home-based exercise improved peak oxygen consumption over 12 weeks in patients with Friedreich’s ataxia compared with placebo and no exercise.
The adjusted between-group difference in change in peak VO2 for combination therapy versus control was 0.21 L/min (95% CI 0.05 to 0.36; padjusted=0.0299), meeting the study’s prespecified efficacy threshold in stage 1 analysis.
