A Phase 3 interventional study of MT1621 in Thymidine Kinase 2 Deficiency, sponsored by Zogenix MDS, Inc.. Withdrawn. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2023-09-01.
Sponsored by Zogenix MDS, Inc. · Phase 3, Interventional, and Treatment
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.
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.
Zogenix MDS, Inc. is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Male and female Participants \<18 years
Drug: MT1621
All patients will receive MT1621 up to a target dose of 400 mg/kg/day each dC and dT, as tolerated.
Also known as: Deoxycytidine (dC) and deoxythymidine (dT)
Proportion of subjects acquiring a Motor Milestone
Proportion of subjects acquiring a motor milestone not present at baseline after 12 months of MT1621 treatment.
Time frame: 12 months
Time to Acquisition of a Motor Milestone
Time to Acquisition of a Motor Milestone that was not present at baseline after 12 months of treatment.
Time frame: 12 months
Survival
Survival after 12 months of treatment
Time frame: 12 months
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is withdrawn, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.
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Zogenix MDS, Inc.