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A study to learn if combination pyrimidine nucleosides is safe, how well it works and how it moves through the bloodstream in participants with thymidine kinase 2 (TK2) deficiency
EU CTIS ID: 2023-510427-29-00
What this study is testing
To characterize the safety and tolerability of continuing treatment with dC/dT (combination pyrimidine nucleosides, administered as doxecitine and doxribtimine) in patients with thymidine kinase 2 (TK2) deficiency previously treated with dC/dT, dCMP/dTMP or doxecitine and doxribtimine
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Signed informed consent by the parent(s) or legally authorized representative (LAR) and/or assent by the study participant (when applicable).
- Confirmed genetic mutation(s) in the TK2 gene.
- Absence of other genetic disease or polygenic disease.
- Current treatment with nucleos(t)ides for TK2 deficiency. Study participants who were not previously enrolled in MT-1621-101 will require Sponsor approval to ensure that collection of clinical and functional measurements prior to treatment are sufficient for evaluating safety and efficacy.
- Female study participants must not be breastfeeding, must have a negative pregnancy test at screening (females ≥10 years old), and have no intention to become pregnant during the course of the study. Female study participants who are of childbearing potential (ie, following menarche until ≥1 year postmenopausal if not anatomically and physiologically incapable of becoming pregnant) must agree and commit to the use of highly effective methods of birth control for the duration of the study and for 30 days after the end of the study. Acceptable methods are defined as those that result, alone or in combination, in a low failure rate (ie, <1% per year) when used consistently and correctly, such as surgical sterilization, an intrauterine device, or hormonal contraception in combination with a barrier method. In certain countries (if permitted by law), women of childbearing potential may instead agree to abide by heterosexual sexual abstinence during the study and for 30 days after the end of the study.
- Male study participants with sexual partners should use condoms for the duration of the study and for 30 days after the last dose of study drug to prevent passing study drug to the partner in the ejaculate. Male participants should be advised not to donate sperm for 30 days after the last dose of study drug.
You likely can't join if
- History of liver disease, or liver function test results (ALT, AST, or total bilirubin) ≥2× upper limit of normal without prior Sponsor approval.
- Other significant medical condition that, in the opinion of the Investigator or Study Sponsor, may confound interpretation of the clinical course of TK2 deficiency.
See the full eligibility criteria
- Signed informed consent by the parent(s) or legally authorized representative (LAR) and/or assent by the study participant (when applicable).
- Confirmed genetic mutation(s) in the TK2 gene.
- Absence of other genetic disease or polygenic disease.
- Current treatment with nucleos(t)ides for TK2 deficiency. Study participants who were not previously enrolled in MT-1621-101 will require Sponsor approval to ensure that collection of clinical and functional measurements prior to treatment are sufficient for evaluating safety and efficacy.
- Female study participants must not be breastfeeding, must have a negative pregnancy test at screening (females ≥10 years old), and have no intention to become pregnant during the course of the study. Female study participants who are of childbearing potential (ie, following menarche until ≥1 year postmenopausal if not anatomically and physiologically incapable of becoming pregnant) must agree and commit to the use of highly effective methods of birth control for the duration of the study and for 30 days after the end of the study. Acceptable methods are defined as those that result, alone or in combination, in a low failure rate (ie, <1% per year) when used consistently and correctly, such as surgical sterilization, an intrauterine device, or hormonal contraception in combination with a barrier method. In certain countries (if permitted by law), women of childbearing potential may instead agree to abide by heterosexual sexual abstinence during the study and for 30 days after the end of the study.
- Male study participants with sexual partners should use condoms for the duration of the study and for 30 days after the last dose of study drug to prevent passing study drug to the partner in the ejaculate. Male participants should be advised not to donate sperm for 30 days after the last dose of study drug.
- Willingness to maintain current treatment regimen and current exercise regimen for the duration of the clinical study.
- 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 (ALT, AST, or total bilirubin) ≥2× upper limit of normal without prior Sponsor approval.
- Other significant medical condition that, in the opinion of the Investigator or Study Sponsor, may confound interpretation of the clinical course of TK2 deficiency.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Israel
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, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Israel. 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 EU CTIS; the study team decides eligibility.