NCT07191912

Confirmatory Study of Govorestat in CMT-SORD

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD)

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 155 (estimated)
• Sponsor: Applied Therapeutics, Inc. · Industry
• Sex: All
• Age: 16 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will assess the effect of govorestat compared to placebo in patients with CMT-SORD for up to 36 months.

Detailed description

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.

Conditions

• Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)

Interventions

Type	Name	Description
DRUG	Govorestat	Govorestat will be provided as a liquid suspension (200mg/mL) for weight-based administration and administered orally at 20 mg/kg QD (every 24 hours)
DRUG	Placebo Control	Placebo will also be provided as a matching liquid suspension to be taken orally QD

Timeline

• Start date: 2025-10-01
• Primary completion: 2028-10-01
• Completion: 2029-01-01
• First posted: 2025-09-25
• Last updated: 2025-09-25

Locations

13 sites across 7 countries: United States, Australia, France, Germany, Italy, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study