Early access
Sign in
WithdrawnPhase 3

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

Key facts

Who runs it
Zogenix MDS, Inc.
Trial code
Status
Withdrawn
Phase
Phase 3
Started
30 Sept 2022
Ended
30 Apr 2025
Type of study
Interventional
Treatments tested
  • MT1621
Record updated
1 September 2023
Why it stopped
Sponsor Decision

Who can take part

Age
Up to 18 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 7 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Current or prior treatment with nucleos(t)ides for TK2d
  • ALT or AST >3 x upper limit of normal and total bilirubin > 2 x ULN or International Normalized Ratio (INR) >1.5
  • Documented clinically significant central nervous system involvement
  • EtCO2>45 mmHg if not on ventilatory support

About the trial

What the trial wants to find out

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.

The group in the trial

  • Single ArmTreatment being tested

    Male and female Participants \<18 years

    Treatment: MT1621

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
A Phase 3b Single Arm Clinical Study to Evaluate the Efficacy and Safety of MT1621 in Nucleos(t)Ide Treatment Naïve Pediatric and Adolescent Subjects With Thymidine Kinase 2 (TK2) Deficiency
Record updated
1 September 2023