Clinical Trial Details
— Status: Withdrawn
Administrative data
NCT number |
NCT04581733 |
Other study ID # |
MT-1621-104 |
Secondary ID |
|
Status |
Withdrawn |
Phase |
Phase 3
|
First received |
|
Last updated |
|
Start date |
September 30, 2022 |
Est. completion date |
April 30, 2025 |
Study information
Verified date |
August 2023 |
Source |
UCB Pharma |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial 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.
Description:
Thymidine kinase 2 (TK2) is a protein involved in the normal function of mitochondria.
Thymidine kinase 2 deficiency (TK2d) is a form of mitochondrial DNA depletion syndrome and is
a very rare inherited genetic disorder. TK2d leads to abnormally low amounts of DNA in
mitochondria and because of this defect, the mitochondria are not able to provide the energy
that cells need to function properly, which causes severe muscle weakness, along with host of
additional symptoms that may involve the respiration, feeding, and ambulation, and can
progress until patients lose many of these abilities. There are no FDA-approved medicines to
treat TK2d.
MT1621 is a therapy that targets the underlying pathophysiology of TK2d by restoring
mitochondrial DNA (mtDNA) replication fidelity. MT1621 consists of a combination of
deoxynucleosides (the building blocks of mtDNA) given orally. Deoxynucleoside combination
therapy improves nucleotide balance, increases mtDNA copy number, improves cell function, and
prolongs life in preclinical models of TK2d.
This is a Phase 3b, prospective, single-arm, multicenter, open-label treatment study to
assess the efficacy and safety of MT1621 in treatment naïve pediatric and adolescent subjects
<18 years of age with TK2d. The study seeks to enroll approximately 16 subjects globally in
this ultra rare disease.