A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects with Friedreich’s Ataxia

Condition studied

Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein, FXN. Friedreich’s ataxia is a rare, progressive, multisystem disease with an incidence that is estimated to be 1:29,000, and a carrier frequency of ~1:85.

Investigational medicinal product(s)

Eligibility

Primary endpoint

PRIMARY: • Difference between participant groups (see Study Design Section) on the Modified Friedreich’s Ataxia Rating Scale (mFARS) total score at study Week 72 compared with baseline

Endpoint detail

SECONDARY: • Difference between participant groups in Clinical Global Impression of Severity (CGI-S) score at study Week 72 compared with baseline, SECONDARY: • Differences between participant groups in scores on physical function tests (Upright Stability Score (USS) Subscale E of the mFARS, 9-hole peg test, and 25-foot walk test) at study Week 72 compared with baseline, SECONDARY: • Differences between participant groups in scores on 1) the Friedreich’s Ataxia Rating Scale – Activities of Daily Living and 2) the fatigue numeric rating scale at study Week 72 compared with baseline, SECONDARY: • Differences between participant groups in left ventricular mass index, measured on echocardiogram, at study Week 72 compared with baseline, SECONDARY: • Differences between participant groups in changes over time in frataxin amount measured in the skin and in the inside of the cheek, SAFETY: • Incidence of side effects and serious side effects happening after receiving nomlabofusp, monitored for the entire study duration, IMMUNOGENICITY: • Incidence of antidrug antibodies (proteins that cause the immune system to fight the drug) throughout the study