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New treatment option for Thymidine Kinase 2 (TK2 ) Deficiency
Official title Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency
ClinicalTrials.gov ID: NCT06754098
What this study is testing
What is Doxecitine and Doxribtimine?
Doxecitine and Doxribtimine is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for thymidine kinase 2 (tk2 ) deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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?
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Signed informed consent by the subject.
- Subject must be greater than 18 years of age at time of consent.
- Genetic diagnosis of TK2 deficiency
- Subject should have evidence of a moderate to severe disease, with motor and or respiratory involvement, shown by one of the following:
- North Star Ambulatory Assessment Scale (NSAA) less than 30
You likely can't join if
- History of liver disease, or liver function test results (alanine aminotransferase [ALT], aspartate transaminase [AST], or total bilirubin) greater...
- Participation in a previous trial of any investigational agent for primary mitochondrial disease within 1 year prior to informed consent, or use of...
- Pregnant (females 10.0 years old or older will have a pregnancy test at screening), or breastfeeding
See the full eligibility criteria
- Signed informed consent by the subject.
- Subject must be greater than 18 years of age at time of consent.
- Genetic diagnosis of TK2 deficiency
- Subject should have evidence of a moderate to severe disease, with motor and or respiratory involvement, shown by one of the following:
- North Star Ambulatory Assessment Scale (NSAA) less than 30
- 6-minute walking test less than 450 meters
- Force Vital Capacity in the sitting position less than 70 percent or a drop in the decubitus position greater than 10 percent or need for mechanical ventilation.
- Disabling symptoms and evidence of motor and/or respiratory function progressive decline.
- Female people must have no intention to become pregnant during the study. Female people who are of childbearing potential (that is, following menarche until at least 1 year post-menopausal if not anatomically and...
- Male people with partners of childbearing potential must agree to use effective contraception methods during the study and for at least 90 days after the last dose of the study medication. Acceptable methods include the...
- Willingness to comply with the study protocol, including but not limited to, all study procedures, study visits, and study drug compliance.
- History of liver disease, or liver function test results (alanine aminotransferase [ALT], aspartate transaminase [AST], or total bilirubin) greater than or equal2 times (2X) the upper limit of normal. Patients with...
- Participation in a previous trial of any investigational agent for primary mitochondrial disease within 1 year prior to informed consent, or use of any other investigational therapy within 30 days (or 3 half-lives...
- Pregnant (females 10.0 years old or older will have a pregnancy test at screening), or breastfeeding
The study team makes the final eligibility decision.
Where it's taking place
- Madrid, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Madrid, Spain. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.