MT1621 for TK2 Deficiency
(Continuation Trial)

Recruiting in Palo Alto (17 mi)

+14 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Zogenix MDS, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

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.

Eligibility Criteria

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

My condition involves a confirmed TK2 gene mutation.

Current treatment with nucleos(t)ides for TK2 deficiency. Patients who were not previously enrolled in MT 1621 101 will require Sponsor approval to ensure that collection of clinical and functional measurements prior to treatment are sufficient to serve as baseline assessments for purposes of evaluating safety and efficacy

I am not pregnant, breastfeeding, or planning to become pregnant and agree to use effective birth control.

+5 more

Exclusion Criteria

I don't have any health issues that could affect my TK2 deficiency study results.

My liver function tests are normal or I have approval for higher levels.

Participant Groups

The safety and effectiveness of two drugs, doxecitine and doxribtimine, are being tested in individuals with thymidine kinase 2 (TK2) deficiency. This Phase 2 trial is open-label, meaning both researchers and participants know which treatment is given.

1Treatment groups

Experimental Treatment

Group I: doxecitine and doxribtimineExperimental Treatment1 Intervention

This is an open label study with all participants in a single arm. Study participants will take doxecitine and doxribtimine up to a maximum of 800 mg/kg/day (400 mg/kg/day doxecitine and 400 mg/kg/day doxiribtimine).