drugset / Trial / NCT04581733
A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2022-09-30
- Primary completion
- 2025-03-31
- Completion
- 2025-04-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | TAS-102 | Small molecule | 400 mg/kg | Oral |
| Subject | deoxythymidine | Unknown | 400 mg/kg | Oral |