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Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency

NCT06754098 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-07-28

No results posted yet for this study

Summary

The purpose of this clinical trial is to evaluate the efficacy and safety of Doxecitin and Doxribtimine (dC+dT) in adult participants with thymidine kinase 2 (TK2) deficiency attended in the Neuromuscular Unit of '12 de Octubre' Hospital.

The main questions it aims to answer are:

* Is dT+dC effective in the treatment of the adult participants with TK2 deficiency?
* Is dT+dC safe in the treatment of adult participants with TK2 deficiency?

Researchers will evaluate the effectiveness of the treatment doxecitin and doxribthymine in adult participants with TK2 deficiency. In addition, the mitochondrial DNA levels before and after treatment (extracted from the muscle and from uroepithelial cells) of these participants will be also studied.

Conditions

  • Thymidine Kinase 2 (TK2 ) Deficiency
  • Mitochondrial Myopathies

Interventions

DRUG

Doxecitine and Doxribtimine

A combination of doxecitine and doxribtimine is administered orally in 3 equal doses given approximately 6 to 8 hours apart .Dose will be increased if the tolerability profile is good.

Sponsors & Collaborators

  • UCB Pharma

    collaborator INDUSTRY

  • Cristina Domínguez González

    lead OTHER

Principal Investigators

  • Cristina D Domínguez González, Dra. · Hospital 12 de Octubre

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-01-01
Primary Completion
2027-01-31
Completion
2027-12-31

Countries

  • Spain

Study Locations

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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 NCT06754098 on ClinicalTrials.gov