NCT03845712

An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency

A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (TK2)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: UCB BIOSCIENCES, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study MT-1621-101 \[NCT03701568\] or who were receiving nucleos(t)ide treatment and were approved by the Sponsor.

Conditions

• Thymidine Kinase 2 Deficiency

Interventions

Type	Name	Description
DRUG	doxecitine and doxribtimine	Doxecitine and doxribtimine is administered orally in 3 equal doses given approximately 6 to 8 hours apart. Doxecitine and doxribtimine is administered with food.

Timeline

• Start date: 2019-07-05
• Primary completion: 2026-06-30
• Completion: 2026-06-30
• First posted: 2019-02-19
• Last updated: 2026-04-13

Locations

8 sites across 3 countries: United States, Israel, Spain

Regulatory

• FDA-regulated drug study