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

NCT04581733 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2023-09-01

No results posted yet for this study

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.

Conditions

  • Thymidine Kinase 2 Deficiency

Interventions

DRUG

MT1621

All patients will receive MT1621 up to a target dose of 400 mg/kg/day each dC and dT, as tolerated.

Sponsors & Collaborators

  • Zogenix MDS, Inc.

    lead INDUSTRY

Principal Investigators

  • UCB Cares · 001 844 599 2273

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-30
Primary Completion
2025-03-31
Completion
2025-04-30
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04581733 on ClinicalTrials.gov