A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
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|ClinicalTrials.gov Identifier: NCT04581733|
Recruitment Status : Not yet recruiting
First Posted : October 9, 2020
Last Update Posted : October 8, 2021
|Condition or disease||Intervention/treatment||Phase|
|Thymidine Kinase 2 Deficiency||Drug: MT1621||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||This is a Phase 3b study. All participants will receive MT1621 up to a target dose of 400 mg/kg/day|
|Masking:||None (Open Label)|
|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|
|Estimated Study Start Date :||November 2021|
|Estimated Primary Completion Date :||March 2025|
|Estimated Study Completion Date :||April 2025|
Experimental: Single Arm
Male and female Participants <18 years
All patients will receive MT1621 up to a target dose of 400 mg/kg/day each dC and dT, as tolerated.
Other Name: Deoxycytidine (dC) and deoxythymidine (dT)
- Proportion of subjects acquiring a Motor Milestone [ Time Frame: 12 months ]Proportion of subjects acquiring a motor milestone not present at baseline after 12 months of MT1621 treatment.
- Time to Acquisition of a Motor Milestone [ Time Frame: 12 months ]Time to Acquisition of a Motor Milestone that was not present at baseline after 12 months of treatment.
- Survival [ Time Frame: 12 months ]Survival after 12 months of treatment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04581733
|Contact: Zogenix Clinical Trials Information Desk||510-920-9700||MT1621ClinStudyInfo@zogenix.com|
|United States, California|
|University of California San Diego Medical Center|
|San Diego, California, United States, 92093|
|Contact: Margo Hammack firstname.lastname@example.org|
|Study Director:||Susan VanMeter, MD||Zogenix, Inc.|