Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency
Recruiting
Phase 2
Interventional Study
Thymidine Kinase 2 (TK2 ) Deficiency
Mitochondrial Myopathies
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- Cristina Domínguez González · Other
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About This Trial
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…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Signed informed consent by the subject.
2. Subject must be greater than 18 years of age at time of consent.
3. Genetic diagnosis of TK2 deficiency
4. Subject should have evidence of a moderate to severe disease, with motor and or respiratory involvement, shown by one of the f…
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