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An Off-Label Single Arm Clinical Study to Evaluate the Efficacy and Safety of doxecitine and doxribtimine in Adult Subjects with Thymidine Kinase 2 (TK2) Deficiency
EU CTIS ID: 2024-510763-35-00
What this study is testing
To evaluate the efficacy of dT+dC in adults with TK2d
- 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 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: a. North Star Ambulatory Assessment Scale (NSAA) < 30 b. 6-minute walking test < 450 meters c. Force Vital Capacity in the sitting position < 70% or a drop in the decubitus position > 10% or need for mechanical ventilation. d. Disabling symptoms and evidence of motor and/or respiratory function progressive decline
- Female subjects must have no intention to become pregnant during the study. Female subjects who are of childbearing potential (ie, following menarche until ≥1 year post-menopausal 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, and be willing to have additional pregnancy tests conducted during 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.
- Male subjects 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 use of condoms combined with spermicidal foam/gel/film/cream/suppository
You likely can't join if
- History of liver disease, or liver function test results (alanine aminotransferase [ALT], aspartate transaminase [AST], or total bilirubin) ≥ 2X ~ upper limit of normal. Patients with transaminases > 2X can participate with the approval and monitoring of a doctor specializing in liver toxicity.
- 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, whichever is longer) prior to informed consent, or participation in other clinical studies, within 30 days prior to informed consent, which in the opinion of the study Sponsor, may potentially confound results from this study.
- Pregnant (females ≥10.0 years old will have a pregnancy test at screening), or breastfeeding.
See the full eligibility criteria
Who can join
- 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: a. North Star Ambulatory Assessment Scale (NSAA) < 30 b. 6-minute walking test < 450 meters c. Force Vital Capacity in the sitting position < 70% or a drop in the decubitus position > 10% or need for mechanical ventilation. d. Disabling symptoms and evidence of motor and/or respiratory function progressive decline
- Female subjects must have no intention to become pregnant during the study. Female subjects who are of childbearing potential (ie, following menarche until ≥1 year post-menopausal 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, and be willing to have additional pregnancy tests conducted during 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.
- Male subjects 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 use of condoms combined with spermicidal foam/gel/film/cream/suppository
- Willingness to comply with the study protocol, including but not limited to, all study procedures, study visits, and study drug compliance.
What rules you out
- History of liver disease, or liver function test results (alanine aminotransferase [ALT], aspartate transaminase [AST], or total bilirubin) ≥ 2X ~ upper limit of normal. Patients with transaminases > 2X can participate with the approval and monitoring of a doctor specializing in liver toxicity.
- 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, whichever is longer) prior to informed consent, or participation in other clinical studies, within 30 days prior to informed consent, which in the opinion of the study Sponsor, may potentially confound results from this study.
- Pregnant (females ≥10.0 years old will have a pregnancy test at screening), or breastfeeding.
The study team makes the final eligibility decision.
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, 65+ years, 18-64 years. The study team makes the final eligibility decision.
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.