Confirmatory Study of Govorestat in CMT-SORD

This international, multi-center, randomized, double-blinded, placebo-controlled, phase 3 study is designed to assess the efficacy and clinical benefit of long-term govorestat administration to patients with CMT-SORD utilizing functional, patient-reported, and clinical outcomes measures including the CMT-HI, 10MWRT, dorsiflexion, the CMT-FOM, and CMT-related lower extremity muscle MRI parameters along with the long-term effect of govorestat on whole blood sorbitol. Long-term safety of govorestat will also be evaluated. Patients (16 to 65 years old) with CMT-SORD will be stratified according to their sex (male vs female) and age before randomization in a 2:1 ratio to govorestat 20 mg/kg once daily (QD) or placebo. There will be a pre-planned Interim Analysis at 2 years (Month 24). If clinical benefit is determined to be met with the Interim Analysis, the study will end and not continue to 3 years. The study will be conducted at up to 16 sites worldwide. Approximately 155 subjects will be enrolled.