Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    MT-1621
Previous Study | Return to List | Next Study

A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04581733
Recruitment Status : Not yet recruiting
First Posted : October 9, 2020
Last Update Posted : October 8, 2021
Sponsor:
Collaborator:
Zogenix, Inc.
Information provided by (Responsible Party):
Modis Therapeutics, Inc.

Brief 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.

Condition or disease Intervention/treatment Phase
Thymidine Kinase 2 Deficiency Drug: MT1621 Phase 3

Detailed 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: N/A
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)
Primary Purpose: Treatment
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

Arm Intervention/treatment
Experimental: Single Arm
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.
Other Name: Deoxycytidine (dC) and deoxythymidine (dT)




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. Survival [ Time Frame: 12 months ]
    Survival after 12 months of treatment



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

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


Contacts
Layout table for location contacts
Contact: Zogenix Clinical Trials Information Desk 510-920-9700 MT1621ClinStudyInfo@zogenix.com

Locations
Layout table for location information
United States, California
University of California San Diego Medical Center
San Diego, California, United States, 92093
Contact: Margo Hammack       mhammack@health.ucsd.edu   
Sponsors and Collaborators
Modis Therapeutics, Inc.
Zogenix, Inc.
Investigators
Layout table for investigator information
Study Director: Susan VanMeter, MD Zogenix, Inc.
Layout table for additonal information
Responsible Party: Modis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04581733    
Other Study ID Numbers: MT-1621-104
First Posted: October 9, 2020    Key Record Dates
Last Update Posted: October 8, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Modis Therapeutics, Inc.:
TK2
TK2d
mitochondrial disorder
mitochondrial disease
Mitochondria
deoxythymidine/deoxythymidine substrate enhancement therapy
dC/dT
deoxythymidine/deoxythymidine
primary mitochondrial myopathy
mitochondrial depletion syndrome
Muscle weakness
Muscle atrophy
Loss of mobility
Thymidine kinase 2 deficiency