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MT1621 for TK2 Deficiency (Continuation Trial)

Phase 2

Waitlist Available

Research Sponsored by Zogenix MDS, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed genetic mutation in the TK2 gene

Absence of other genetic disease or polygenic disease

Must not have

History of liver disease, or liver function test results (ALT, AST, or total bilirubin) ≥2× upper limit of normal without prior Sponsor approval

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug, MT1621, for safety and efficacy in people with a deficiency in the TK2 protein. The trial will compare measurements taken before treatment with dC/dT (a similar drug) to those taken after treatment with MT1621.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects aren't listed here, common ones may include reactions at the injection site, fatigue, nausea or digestive issues. Participants will be monitored closely for any adverse effects throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My condition involves a confirmed TK2 gene mutation.

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I do not have any genetic or complex inherited diseases.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety as adverse events (AEs): number of participants who experience adverse events

Safety as determined by electrocardiograms (ECGs)

Safety as determined by laboratory measurements

Secondary study objectives

AUC - area under the plasma concentration time curve of deoxycytidine and deoxythymidine

Apparent elimination half-life (t1/2) of deoxycytidine and deoxythymidine

Biomarkers (plasma from blood)

+7 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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).

0 / 3Eligibility criteria met