MedTrace Logo

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

NCT03845712 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 47

Last updated 2026-05-01

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • UCB BIOSCIENCES, Inc.

    lead INDUSTRY

Principal Investigators

  • UCB Cares · 001 844 599 2273

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-05
Primary Completion
2026-06-30
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States
  • Israel
  • Spain

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03845712 on ClinicalTrials.gov