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)
Applied Therapeutics, Inc.
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.
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 t…
Eligibility
- Age range
- 16–65 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Willing and able to provide signed and dated informed consent prior to any study-related procedures and willing and able to comply with all study procedures. * Male and non-pregnant, non-lactating female patients between the ages of 16 and 65 years, inclusive. * Females must be of non-childbearing potential (defined as surgically sterile \[i.e., had a bilateral tubal ligation, hysterectomy, or bilateral oophorectomy ≥6 months prior to the first dose of study drug\] or postmenopausal for ≥1 year \[confirmatory follicle stimulating hormone or FSH test results required\] pr…
Interventions
- DrugGovorestat
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)
- DrugPlacebo Control
Placebo will also be provided as a matching liquid suspension to be taken orally QD
Locations (13)
- University of IowaIowa City, Iowa
- Clinic for Special ChildrenGordonville, Pennsylvania
- Sydney ChildrensSydney
- CHU La TimoneMarseille
- Institut de MyologieParis
- Uniklinik of the RWTH Aachen UniversityAachen