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

StatusRecruiting
PhasePhase 2
Started
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Record History

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  1. Notable Primary endpoints: details revised at 1 of 10 entries
    • Eligibility criteria expanded, about 14 words
    • Site details revised
    • Publications: details revised at 4 of 4 entries
  2. Notable Enrollment target: 5 to 15
    • Eligibility criteria expanded, about 11 words
    • Study description expanded, about 2 words
    v4
  3. Minor Site details revisedv3
  4. Notable Trial status: Not Yet Recruiting to Recruitingv2 Show 4 more changesShow less
    • Site details revised
    • Publications: 3 to 4
    • Study title shortened, about 13 words
    • Study description expanded, about 1 word
  5. Minor Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06754098
Lead Sponsor Cristina Domínguez González
Collaborators: UCB Pharma
Conditions
(from sponsor)
Thymidine Kinase 2 (TK2 ) Deficiency, Mitochondrial Myopathies
Related terms Mitochondrial Myopathies
Enrollment 15 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov