WithdrawnPhase 3

A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)

Stopped: Sponsor Decision

0Started 2022-09-30

Plain-language summary

This is a Phase 3b, prospective, single-arm, multicenter, open-label treatment study of the efficacy and safety of MT1621 in pediatric and adolescent patients with thymidine kinase 2 deficiency (TK2d). In order to be eligible for this study, participants must have genetic confirmation of TK2d and must not have ever received MT1621 or nucleos(t)ides before entering the study.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Subject must be aged birth to \<18 years of age on the day of consent. * Diagnosis of TK2 deficiency based on confirmed disease-causing mutation(s) in the TK2 gene. * Onset of TK2d at ≤12 years of age as defined as the age at which the first TK2d symptom occurred. Exclusion Criteria: * Documented clinically significant central nervous system involvement. * ALT or AST \>3 x upper limit of normal and total bilirubin \> 2 x ULN or International Normalized Ratio (INR) \>1.5. * EtCO2\>45 mmHg if not on ventilatory support * Current or prior treatment with nucleos(t)ides for TK2d.

What they're measuring

Proportion of subjects acquiring a Motor Milestone

Timeframe: 12 months

Trial details

NCT IDNCT04581733

SponsorZogenix MDS, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-03-31

View on ClinicalTrials.gov More Thymidine Kinase 2 Deficiency trials