Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome

PHASE2RecruitingINTERVENTIONAL
Enrollment

200

Participants

Timeline

Start Date

October 18, 2021

Primary Completion Date

June 30, 2029

Study Completion Date

December 30, 2029

Conditions
Mitochondrial DiseasesMitochondrial EncephalomyopathyMitochondrial EncephalopathyMitochondrial DNA DepletionMitochondrial Metabolism Disorders
Interventions
COMBINATION_PRODUCT

deoxycytidine and deoxythymidine

The Investigational Product (IP) dC/dT100-400 will be administered orally every day (QD) and the dose is divided over trid or bid for the daily dose of 100 mg/kg from Day 1-7, 200 mg/kg from Day 8-14, 300 mg/kg from Day 15- 21 and 400 mg/kg from Day 22 to 1825. Doses was chosen according to the safety and efficacy doses used in the literature.

Trial Locations (1)

H4A 3J1

RECRUITING

Research InstituMcGill University Health Centre - Children Hospital of Montreal, Montreal

All Listed Sponsors
lead

Kenneth Myers, MD

OTHER