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

000, and a carrier frequency of ~1:85., 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, multisystem disease with an incidence that is estimated to be 1:29, progressive

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

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

No related papers found yet.