A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
Sponsored by Zogenix MDS, Inc.
About this trial
Last updated 2 years ago
Study ID
MT-1621-104
Status
Withdrawn
Type
Interventional
Phase
Phase 3
Placebo
No
Accepting
Up to 18 Years
All
Not accepting
Healthy Volunteers
Trial Timing
Ended 2 months ago
What is this trial about?
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.
What are the participation requirements?
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.