Signed informed consent by the parent(s) or legally authorized representative (LAR) and/or assent by the patient (when applicable).
Confirmed genetic mutation in the TK2 gene.
Absence of other genetic disease or polygenic disease.
Current treatment with nucleos(t)ides for TK2 deficiency. Patients who were not previously enrolled in MT 1621 101 will require Sponsor approval to ensure that collection of clinical and functional measurements prior to treatment are sufficient to serve as baseline assessments for purposes of evaluating safety and efficacy.
Female patients must not be breastfeeding, have a negative pregnancy test at screening (females ≥10 years old), and have no intention to become pregnant during the course of the study. Female patients who are of childbearing potential (ie, following menarche until ≥1 year post-menopausal if not anatomically and physiologically incapable of becoming pregnant) must agree and commit to the use of highly effective methods of birth control for the duration of the study and for 30 days after the end of the study. Acceptable methods are defined as those that result, alone or in combination, in a low failure rate (ie, <1% per year) when used consistently and correctly, such as surgical sterilization, an intrauterine device, or hormonal contraception in combination with a barrier method. In certain countries (if permitted by law), women of childbearing potential may instead agree to abide by heterosexual sexual abstinence during the study and for 30 days after the end of the study.
Male patients with sexual partners should use condoms for the duration of the study and for 30 days after the last dose of study drug to prevent passing study drug to the partner in the ejaculate. Male participants should be advised not to donate sperm for 30 days after the last dose of study drug.
Willingness to maintain current treatment regimen and current exercise regimen for the duration of the clinical study.
Willingness to comply with the study protocol, including but not limited to, all study procedures, study visits, and study drug compliance.
Exclusion Criteria:
History of liver disease, or liver function test results (ALT, AST, or total bilirubin) ≥2× upper limit of normal without prior Sponsor approval.
Other significant medical condition that, in the opinion of the Investigator or Study Sponsor, may confound interpretation of the clinical course of TK2 deficiency.
Source : Importé depuis le centre
Cohortes
Thérapie ou Intervention proposée
Cohortes
Nom
Condition médicale
Traitement
État du recrutement
MT1621, dC/dT
Donnée non disponible
This is an open label study with all participants in a single arm. Patients will take MT1621 up to a maximum of 800 mg/kg/day (400 mg/kg/day dC and 400 mg/kg/day dT). MT1621 is deoxycytidine (dC) and deoxythymidine (dT) powders for solution for reconstitution in water or apple juice. Study drug will be supplied as powder in packets containing 0.5 or 2.0 g or 4.0 g of dC or dT, and is typically dosed three times/day. MT1621 should be administered with food.
Inconnu
MT1621, dC/dT
État du recrutement
unknown
This is an open label study with all participants in a single arm. Patients will take MT1621 up to a maximum of 800 mg/kg/day (400 mg/kg/day dC and 400 mg/kg/day dT). MT1621 is deoxycytidine (dC) and deoxythymidine (dT) powders for solution for reconstitution in water or apple juice. Study drug will be supplied as powder in packets containing 0.5 or 2.0 g or 4.0 g of dC or dT, and is typically dosed three times/day. MT1621 should be administered with food.
Données à jour depuis :
2 septembre 2023
Description de l'étude
Description de l'étude
Résumé de l'étude
This is a Phase 2 prospective, open-label treatment study of the safety and efficacy of MT1621 in TK2 deficient subjects who participated in the retrospective Study MT-1621-101. Subjects who are being treated with dC/dT and did not participate in MT-1621-101 may also be allowed to enroll with Sponsor approval. For all subjects, it is important to ensure that collection of clinical and functional measurements prior to treatment with dC/dT are sufficient to serve as baseline assessments for purposes of evaluating safety and efficacy.
Source : Importé depuis le centre
This is a Phase 2 prospective, open-label treatment study of the safety and efficacy of MT1621 in TK2 deficient subjects who participated in the retrospective Study MT-1621-101. Subjects who are being treated with dC/dT and did not participate in MT-1621-101 may also be allowed to enroll with Sponsor approval. For all subjects, it is important to ensure that collection of clinical and functional measurements prior to treatment with dC/dT are sufficient to serve as baseline assessments for purposes of evaluating safety and efficacy.
In MT-1621-102, all subjects will be treated with MT1621 at one of 3 protocol-specified dose levels: 260 mg/kg/day (130 mg/kg/day dC and 130 mg/kg/day dT), 520 mg/kg/day (260 mg/kg/day dC and 260 mg/kg/day dT), or 800 mg/kg/day (400 mg/kg/day dC and 400 mg/kg/day dT), divided in three equal doses (tid). On enrollment into the study, subjects already taking their medication at one of these dose levels will either transition from chemical-grade dC/dT or dCMP/dTMP to the same dose of MT1621, or continue use of MT1621 at their current dose. Subjects who were previously taking chemical-grade dC/dT, dCMP/dTMP, or MT1621 at a dose other than one of these dose levels will be transitioned to a protocol-specified dose of MT1621, whichever is closest to the subjects' previous dose of chemical-grade dC/dT, chemical-grade dCMP/dTMP, or MT1621. The dose may be reduced for tolerability reasons.
Safety and efficacy will be assessed upon enrollment, at 1 month, every 3 months through 18 months, every 6 months through 36 months, then annually thereafter, and at end of study participation. For subjects who did not participate in Study MT-1621-101, specific assessments for each subject will be determined by the Sponsor in discussion with the Investigator based on data collected as baseline assessments for purposes of evaluating safety and efficacy. MT1621 should be administered with food.
The study will evaluate sparse PK sampling at steady state using sparse sampling methodology.
Source : Importé depuis le centre
Centres participants
Sites
Centres participants
10
affichés
sur
15
centres
HOSPITAL 12 DE OCTUBRE
Madrid
SPAIN
Recrutement local
État du recrutement:
FERMÉ
HOSPITAL UNIVERSITARIO VIRGEN DEL ROCIO
Seville
SPAIN
Recrutement local
État du recrutement:
FERMÉ
HOSPITAL VALL D'HEBRON
Barcelona
SPAIN
Recrutement local
État du recrutement:
FERMÉ
NEW YORK PRESBYTERIAN HOSPITAL-COLUMBIA UNIVERSITY MEDICAL CENTER
New york
NEW YORK, UNITED STATES
Recrutement local
État du recrutement:
FERMÉ
RAMBAM HOSPITAL
Haifa
ISRAEL
Recrutement local
État du recrutement:
FERMÉ
SANT JOAN DE DEU HOSPITAL
Barcelona
SPAIN
Recrutement local
État du recrutement:
FERMÉ
TK0102 1005
New york
NEW YORK, UNITED STATES
Recrutement local
État du recrutement:
FERMÉ
TK0102 3031
Madrid
SPAIN
Recrutement local
État du recrutement:
FERMÉ
TK0102 3101
Sevilla
SPAIN
Recrutement local
État du recrutement:
FERMÉ
TK0102 3102
Barcelona
SPAIN
Recrutement local
État du recrutement:
FERMÉ
Centres participants
Source d'information
Dernière modification :
2 septembre 2023
Données à jour depuis :
4 mai
Origine des données :
clinicaltrials.gov
