MT1621 for TK2 Deficiency
(Continuation Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study MT-1621-101 \[NCT03701568\] or who were receiving nucleos(t)ide treatment and were approved by the Sponsor.
Will I have to stop taking my current medications?
The trial requires participants to continue their current treatment with nucleos(t)ides for TK2 deficiency throughout the study.
Is MT1621 safe for humans?
How does the drug MT1621 differ from other treatments for TK2 deficiency?
MT1621 is unique because it uses a combination of deoxycytidine and deoxythymidine monophosphates to bypass the deficiency in the TK2 enzyme, which helps restore balance in the building blocks needed for mitochondrial DNA. This approach is the first effective pharmacologic treatment for TK2 deficiency, significantly improving symptoms and extending lifespan in preclinical models.12345
Who Is on the Research Team?
UCB Cares
Principal Investigator
001 844 599 2273
Are You a Good Fit for This Trial?
This trial is for patients with TK2 deficiency who have a confirmed genetic mutation in the TK2 gene, are not pregnant or breastfeeding, agree to use contraception, and can maintain their current treatment and exercise regimens. They must not have liver disease or certain other medical conditions that could affect study results.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive doxecitine and doxribtimine up to a maximum of 800 mg/kg/day
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- MT1621
Find a Clinic Near You
Who Is Running the Clinical Trial?
Zogenix MDS, Inc.
Lead Sponsor
Modis Therapeutics, Inc.
Lead Sponsor
UCB BIOSCIENCES, Inc.
Lead Sponsor
Zogenix, Inc.
Industry Sponsor