Confirmatory Study of Govorestat in CMT-SORD

PHASE3Not yet recruitingINTERVENTIONAL
Enrollment

155

Participants

Timeline

Start Date

October 31, 2025

Primary Completion Date

October 31, 2028

Study Completion Date

January 31, 2029

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

Trial Locations (13)

2013

Sydney Childrens, Sydney

10117

Charité - Universitätsmedizin Berlin, Berlin

13005

CHU La Timone, Marseille

17529

Clinic for Special Children, Gordonville

20133

Carlo Besta Neurological Institute, Milan

34010

Koç University Hospital, Istanbul

34093

Istanbul University, Istanbul

37075

University Medicine Gottingen (UMG), Göttingen

46026

La Fe University and Polytechnic Hospital, Valencia

52074

Uniklinik of the RWTH Aachen University, Aachen

52242

University of Iowa, Iowa City

75013

Institut de Myologie, Paris

08035

Vall d'Hebron Institut de Recerca (VHIR), Barcelona

Sponsors
All Listed Sponsors
lead

Applied Therapeutics, Inc.

INDUSTRY