MT1621 for TK2 Deficiency
(Continuation Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the safety and effectiveness of a treatment called MT1621 for individuals with TK2 deficiency, a condition affecting cellular energy production and potentially causing muscle weakness. Participants will receive two experimental drugs, doxecitine and doxribtimine, to assess their impact on symptoms. Suitable candidates for this trial include those already receiving treatment for TK2 deficiency and with a confirmed genetic mutation in the TK2 gene. As a Phase 2 trial, the research focuses on evaluating the treatment's efficacy in an initial, smaller group of participants.
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 there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that the combination of doxecitine and doxribtimine is being tested for safety in people with thymidine kinase 2 deficiency, a rare condition. Previous studies have used this combination without major safety issues. One study examined how the body processes these drugs and found they were generally well-tolerated.
Specific safety data from these studies is not available, but the Phase 2 trial status indicates the treatment has already passed initial safety tests. This suggests major safety concerns are less likely, yet the treatment remains under close monitoring to ensure participant safety.12345Why are researchers excited about this trial's treatments?
Most treatments for TK2 deficiency focus on managing symptoms and providing supportive care because there aren't many targeted therapies available. But MT1621 stands out because it combines doxecitine and doxribtimine, which are designed to boost energy production in cells by targeting mitochondrial function directly. This approach is different from standard treatments, which don't address the root cause of the energy deficiency. Researchers are excited about MT1621 because it has the potential to improve muscle strength and reduce the progression of this rare and debilitating condition by tackling the underlying mitochondrial dysfunction.
What evidence suggests that MT1621 might be an effective treatment for TK2 deficiency?
Research shows that the combination of doxecitine and doxribtimine, which participants in this trial will receive, can greatly benefit people with thymidine kinase 2 (TK2) deficiency. Studies have found a 95% reduction in the risk of death for patients who received this treatment, significantly lowering the chance of dying from TK2 deficiency. Results also indicate an estimated 85% to 93% decrease in the risk of death from the onset of symptoms. These findings suggest that this treatment could be highly effective for patients with TK2 deficiency.12356
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