Thymine Kinase 2 DeficiencyNCT06590493
Doxecitine and Doxribtimine-Expanded Access
A study testing doxecitine and doxribtimine in people with Thymine Kinase 2 Deficiency, run by UCB BIOSCIENCES, Inc..
Trial summary
This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status: TK0113: Available TK0115: No longer Available Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thymine Kinase 2 Deficiency
- What is being tested?
- doxecitine and doxribtimine
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- TK0113:
- Pediatric and adult patients with a diagnosis of TK2d based on confirmed reportable variant(s) in the TK2 gene in countries where UCB has an affiliate/local safety officer Signs and symptoms compatible with TK2d disease
- Risk of major disability or death resulting from TK2d
- The patient must be willing to receive treatment with doxecitine and doxribtimine via this program, which includes signing an authorization form for sharing genetic test results, medical data and other related information with UCB, its third-party agents and health authorities
- The patient/legal guardian or representative has given informed consent (and age-appropriate assent) to treatment prior to administering doxecitine and doxribtimine in a manner consistent with all national requirements. This also includes consent for the transmission of a copy of the anonymized data, such as serious adverse event (SAE) and pregnancy reports (in compliance with local regulatory authority requirements) to UCB third-party agents where allowable by local regulations and to the country regulatory authority as required.
- The patient does not qualify for or is unable to participate in a UCB sponsored ongoing clinical trial evaluating doxecitine and doxribtimine.
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- TK0115:
- Diagnosis of TK2d based on confirmed reportable variant(s) in the TK2 gene
- Age of TK2d symptom onset ≤ 12 years
- The patient or care provider must be willing to receive treatment with doxecitine and doxribtimine via this program
- Patient has consented to the contraception, pregnancy, and lactation requirements where relevant
- The patient does not qualify for or is unable to participate in a UCB-sponsored clinical trial evaluating doxecitine and doxribtimine for the treatment of patients with TK2d
Who cannot join
- TK0113:
- Confirmed diagnosis of other genetic or polygenic disease likely to confound clinical presentation of TK2 deficiency
- Patient has a hypersensitivity to any of the excipients in doxecitine and doxribtimine
- Inability to tolerate oral or gastric tube administration of doxecitine and doxribtimine
- History of liver disease, or liver function test results (alanine aminotransferase[ALT], aspartate transaminase [AST], or total bilirubin) ≥3× upper limit of normal at Screening without prior Sponsor approval
- Patients with elevated transaminases (ALT, AST > 3xupper limit of normal) without increase in bilirubin should be further evaluated to exclude other causes of liver injury and may be enrolled with UCB approval or may be rescreened
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- Renal insufficiency requiring dialysis
- Discontinuation of prior nucleos(t)ide treatment for TK2d because of adverse event(s)
- Any other concurrent inborn errors of metabolism
- Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis
- Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the patient or compromise his/her ability to comply with, or adversely impact, protocol requirements
- Concurrent participation in another interventional trial or named patient program where investigational drug is received
- Any other reason that UCB may determine that the patient is unsuitable for named patient / compassionate use of doxecitine and doxribtimine
- TK0115:
- History of liver disease or liver function test results (alanine aminotransferase [ALT], aspartate transaminase [AST], or total bilirubin) ≥ 3 × upper limit of normal (ULN) at Baseline or bilirubin > 1.5 × ULN. Isolated bilirubin > 1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%
- Patients with elevated transaminases (ALT and/or AST > 3 × ULN) without increase in bilirubin should be further evaluated to exclude other causes of liver injury and may be enroled with UCB approval
- Patient has a medical, mental, or any other extenuating condition or circumstance that may, in the opinion of the treating physician, pose an undue safety risk to the patient or compromise his/her ability to comply with, or adversely impact, protocol requirements
- Renal insufficiency requiring dialysis
- Discontinuation of prior nucleoside treatment for TK2d due to adverse event(s) (AEs)
- Concurrent participation in another interventional trial or named patient program where investigational drug is received
- Known hypersensitivity to doxecitine and doxribtimine or any excipients of the drug substance (silicon dioxide and magnesium stearate)
- Inability to tolerate oral or enteral feeding tube administration of doxecitine and doxribtimine
- Female patients will not be eligible to participate if they are pregnant, plan to become pregnant during the course of the program, or are breastfeeding
Trial locations and contacts
No sites are listed yet.
About this study
This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status:
- TK0113: Available
- TK0115: No longer Available
Read the detailed description
- TK0113: Data Collection for Patients in the Company Supported Expanded Access Program
- TK0115: TREATMENT PROTOCOL FOR DOXECITINE AND DOXRIBTIMINE - Company-sponsored Intermediate-size Cohort Expanded Access Program for Doxecitine and Doxribtimine for Patients with Thymidine Kinase 2 Deficiency (TK2d) with an Age of Symptom Onset ≤ 12 Years
- Conditions
- Thymine Kinase 2 Deficiency
- Being tested
- doxecitine and doxribtimine
- Keywords
- Thymine Kinase 2 Deficiency, TK2, TK2d
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
doxecitine and doxribtimineDrug
TK0113 and TK0115: Doxecitine and doxribtimine is dosed daily and will be administered orally or via feeding tube. The patient may continue to receive doxecitine and doxribtimine treatment within the EAP as long as the benefit / risk balance remains positive in the opinion of the treating physician or access to commercially available doxecitine and doxribtimine, when it becomes available.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thymine Kinase 2 DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- UCB BIOSCIENCES, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06590493
- Sponsor's protocol ID
- doxecitine and doxribtimine-EA
- Status last verified
- Jun 2026
- First submitted
- 5 Sep 2024
- Last updated
- 5 Jun 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Doxecitine and Doxribtimine-Expanded Access
- ClinicalTrials.gov ID
- NCT06590493
- Acronym / sponsor ID
- doxecitine and doxribtimine-EA
- Conditions
- Thymine Kinase 2 Deficiency
- Keywords
- Thymine Kinase 2 Deficiency, TK2, TK2d
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- UCB BIOSCIENCES, Inc.Industry (pharma, biotech, devices)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status:
- TK0113: Available
- TK0115: No longer Available
Detailed description
- TK0113: Data Collection for Patients in the Company Supported Expanded Access Program
- TK0115: TREATMENT PROTOCOL FOR DOXECITINE AND DOXRIBTIMINE - Company-sponsored Intermediate-size Cohort Expanded Access Program for Doxecitine and Doxribtimine for Patients with Thymidine Kinase 2 Deficiency (TK2d) with an Age of Symptom Onset ≤ 12 Years
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Pediatric and adult patients with a diagnosis of TK2d based on confirmed reportable variant(s) in the TK2 gene in countries where UCB has an affiliate/local safety officer Signs and symptoms compatible with TK2d disease
- Risk of major disability or death resulting from TK2d
- The patient must be willing to receive treatment with doxecitine and doxribtimine via this program, which includes signing an authorization form for sharing genetic test results, medical data and other related information with UCB, its third-party agents and health authorities
- The patient/legal guardian or representative has given informed consent (and age-appropriate assent) to treatment prior to administering doxecitine and doxribtimine in a manner consistent with all national requirements. This also includes consent for the transmission of a copy of the anonymized data, such as serious adverse event (SAE) and pregnancy reports (in compliance with local regulatory authority requirements) to UCB third-party agents where allowable by local regulations and to the country regulatory authority as required.
- The patient does not qualify for or is unable to participate in a UCB sponsored ongoing clinical trial evaluating doxecitine and doxribtimine.
Key exclusion criteria (23 listed)
- Confirmed diagnosis of other genetic or polygenic disease likely to confound clinical presentation of TK2 deficiency
- Patient has a hypersensitivity to any of the excipients in doxecitine and doxribtimine
- Inability to tolerate oral or gastric tube administration of doxecitine and doxribtimine
- History of liver disease, or liver function test results (alanine aminotransferase[ALT], aspartate transaminase [AST], or total bilirubin) ≥3× upper limit of normal at Screening without prior Sponsor approval
- Patients with elevated transaminases (ALT, AST > 3xupper limit of normal) without increase in bilirubin should be further evaluated to exclude other causes of liver injury and may be enrolled with UCB approval or may be rescreened
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| doxecitine and doxribtimineTK0113 and TK0115: Doxecitine and doxribtimine is dosed daily and will be administered orally or via feeding tube. The patient may continue to receive doxecitine and doxribtimine treatment within the EAP as long as the benefit / risk balance remains positive in the opinion of the treating physician or access to commercially available doxecitine and doxribtimine, when it becomes available. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273
Questions about this trial
What is this clinical trial studying?
A study testing doxecitine and doxribtimine in people with Thymine Kinase 2 Deficiency, run by UCB BIOSCIENCES, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact UCB Cares (1-844-599-2273 (USA), UCBCares@ucb.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2026.