Treatment of TK2 Deficiency With Thymidine and Deoxycytidine

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

May 16, 2017

Primary Completion Date

May 31, 2026

Study Completion Date

May 31, 2026

Conditions
Mitochondrial DNA Depletion Syndrome 2 Myopathic TypeThymidine Kinase 2 Deficiency
Interventions
DRUG

Thymidine

Mitochondrial DNA nucleotide precursors. Dose escalation: 130mg/kg/day x 14 days, 260 mg/kg/day x 14 days, and 400mg/kg/day as tolerated. Compounds are taken orally and divided into 3 doses daily.

Trial Locations (1)

10032

Columbia University Irving Medical Center, New York

All Listed Sponsors
collaborator

Muscular Dystrophy Association

OTHER

collaborator

Hospital Universitario 12 de Octubre

OTHER

collaborator

Instituto de Salud Carlos III

OTHER_GOV

collaborator

University of Seville

OTHER

collaborator

Medical Research Council Mitochondrial Biology Unit

UNKNOWN

collaborator

Centro de Investigación Biomédica en Red Enfermedades Raras (CIBERER), Spain

UNKNOWN

collaborator

Hospitales Universitarios Virgen del Rocío

OTHER

collaborator

Universitat Autonoma de Barcelona

OTHER

lead

Columbia University

OTHER

NCT03639701 - Treatment of TK2 Deficiency With Thymidine and Deoxycytidine | Biotech Hunter | Biotech Hunter