Doxecitine and Doxribtimine-Expanded Access
ConditionThymine Kinase 2 Deficiency
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
AgeNot listed
SponsorUCB BIOSCIENCES, Inc.
About this trial
This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status:
* TK0113: Available * TK0115: No longer Available
Eligibility criteria
Qualifiers
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.
Disqualifiers
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
Trial design
Treatments tested in this trial
- doxecitine and doxribtimine
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Source ClinicalTrials.gov