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    Summary
    EudraCT Number:2018-004277-27
    Sponsor's Protocol Code Number:MT-1621-102
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-04-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-004277-27
    A.3Full title of the trial
    A Phase 2 open-label study of continuation treatment with combination pyrimidine nucleos(t)ides in patients with thymidine kinase 2 deficiency (TK2)
    Estudio abierto en fase II del tratamiento de continuación con una combinación de nucleós(t)idos de pirimidina en pacientes con deficiencia de timidina cinasa 2 (TK2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to test the safety and effectiveness of MT1621 in people who are deficient in an enzyme located in the mitochondrial called tk2.
    Estudio para comprobar la seguridad y eficacia de MT1621 en personas con deficiencia de una enzima mitocondrial denominada tk2
    A.4.1Sponsor's protocol code numberMT-1621-102
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorModis Therapeutics, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportModis Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationModis Therapeutics, Inc.
    B.5.2Functional name of contact pointClinical Study Team
    B.5.3 Address:
    B.5.3.1Street Address409 13th Street, Suite 700
    B.5.3.2Town/ cityOakland
    B.5.3.3Post codeCA 94612
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1510925 1315
    B.5.6E-mailinfo@modistx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1870
    D.3 Description of the IMP
    D.3.1Product namedeoxycytidine and deoxythymidine
    D.3.2Product code MT1621
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMT1621
    D.3.9.2Current sponsor codeMT1621
    D.3.9.3Other descriptive namedeoxycytidine and deoxythymidine
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Thymidine kinase 2 deficiency
    Deficiencia de timidina cinasa 2
    E.1.1.1Medical condition in easily understood language
    Genetic disease in which patients' cells have reduced amounts of mitochondrial DNA leading to progressive muscle weakness.
    Enfermedad genética en la cual las células del paciente contienen cantidades reducidas de DNA mitocondrial que conducen a una debilidad muscular progresiva.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To characterize the safety profile of continuing treatment with dC/dT (combination pyrimidine nucleosides, administered as MT1621) in patients with thymidine kinase 2 (TK2) deficiency previously treated with dC/dT and/or dCMP/dTMP
    Caracterizar el perfil de seguridad de la continuación del tratamiento con dC/dT (combinación de nucleósidos de pirimidina, administrado como MT1621) en pacientes con deficiencia de timidina cinasa 2 (TK2) tratados previamente con dC/dT y/o dCMP/dTMP
    E.2.2Secondary objectives of the trial
    • To determine the continued efficacy of dC/dT (administered as MT1621) in patients with TK2 deficiency compared with data recorded for these patients prior to treatment with pyrimidine nucleos(t)ides
    • To determine the effects of pyrimidine nucleos(t)ides on clinical parameters associated with TK2 deficiency
    • To determine the pharmacokinetics (PK) of MT1621 using sparse sampling methodology
    • To explore the effects of disease and/or MT1621 on proposed disease and/or treatment-related biomarkers
    • To characterize the quality of life of patients with TK2 deficiency
    • To characterize the health care utilization of patients with TK2 deficiency
    • Determinar la eficacia continuada de dC/dT (administrado como MT1621) en pacientes con deficiencia de TK2 en comparación con los datos registrados para estos pacientes antes del tratamiento con nucleós(t)idos de pirimidina
    • Determinar los efectos de los nucleós(t)idos de pirimidina sobre los parámetros clínicos asociados con la deficiencia de TK2
    • Determinar la farmacocinética (FC) de MT1621 utilizando la metodología de recogida dispersa de muestras
    • Explorar los efectos de la enfermedad y/o MT1621 sobre los biomarcadores de la enfermedad y/o relacionados con el tratamiento que se proponen
    • Caracterizar la calidad de vida de los pacientes con deficiencia de TK2
    • Caracterizar la utilización de los recursos sanitarios de los pacientes con deficiencia de TK2
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent by the parent(s) or legally authorized representative (LAR) and/or assent by the patient (when applicable).
    2. Confirmed genetic mutation in the TK2 gene.
    3. Absence of other genetic disease or polygenic disease.
    4. 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.
    5. 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.
    6. Willingness to maintain current treatment regimen and current exercise regimen for the duration of the clinical study.
    7. Willingness to comply with the study protocol, including but not limited to, all study procedures, study visits, and study drug compliance.
    1. Consentimiento informado firmado por el/los progenitor(es) o el representante legal autorizado (RLA) y/o asentimiento del paciente (cuando corresponda).
    2. Confirmación de la mutación genética en el gen TK2.
    3. Ausencia de otra enfermedad genética o enfermedad poligénica.
    4. Tratamiento actual con nucleós(t)idos para la deficiencia de TK2. Los pacientes que no estaban inscritos anteriormente en MT-1621-101 necesitará la aprobación del promotor para garantizar que la obtención de las mediciones clínicas y funcionales antes del tratamiento son suficientes para servir como evaluaciones iniciales para los fines de evaluar la seguridad y la eficacia.
    5. Las pacientes de sexo femenino no deben estar en periodo de lactancia, deben presentar una prueba de embarazo negativa en la selección (mujeres ≥10 años de edad) y no tener intención de quedarse embarazadas durante el transcurso del estudio. Las pacientes de sexo femenino que estén en edad fértil (es decir, tras la menarquia y hasta ≥1 año después de la menopausia, si no son anatómica y fisiológicamente incapaces de quedarse embarazadas) deben aceptar y comprometerse a usar métodos anticonceptivos altamente eficaces durante todo el estudio y durante 30 días después del final del estudio. Los métodos aceptables se definen como aquellos que, solos o en combinación, presentan una tasa de fallo baja (es decir, <1 % por año) cuando se usan de forma constante y correcta, como, por ejemplo, esterilización quirúrgica, un dispositivo intrauterino, o un anticonceptivo hormonal en combinación con un método de barrera. En determinados países (si lo permite la ley), las mujeres en edad fértil pueden, en su lugar, optar por la abstinencia de relaciones sexuales heterosexuales durante el estudio y durante 30 días después del final del estudio.
    6. Disposición para mantener la pauta de tratamiento actual y el régimen de ejercicio actual mientras dure el estudio clínico.
    7. Disposición para cumplir con el protocolo del estudio, incluidos, entre otros, todos los procedimientos del estudio, las visitas del estudio y el cumplimiento con el fármaco del estudio.
    E.4Principal exclusion criteria
    1. History of liver disease, or liver function test results (ALT, AST, or total bilirubin) ≥2× upper limit of normal without prior Sponsor approval.
    2. Other significant medical condition that, in the opinion of the Investigator or Study Sponsor, may confound interpretation of the clinical course of TK2 deficiency.
    1. Antecedentes de enfermedad hepática, o resultados de las pruebas de función hepática (ALT, AST o bilirrubina total) ≥2 × límite superior de la normalidad, sin la aprobación previa del promotor.
    2. Otra afección médica importante que, en opinión del investigador o del promotor del estudio, pueda confundir la interpretación de la evolución clínica de la deficiencia de TK2.
    E.5 End points
    E.5.1Primary end point(s)
    Safety, as determined by adverse events (AE), laboratory measurements, and electrocardiograms
    Seguridad, determinada por los acontecimientos adversos (AA), las mediciones analíticas y los electrocardiogramas
    E.5.1.1Timepoint(s) of evaluation of this end point
    See Schedule of Assessments
    Ver calendario de evaluaciones
    E.5.2Secondary end point(s)
    • Efficacy endpoints may include the following:
    - motor function assessments (eg, achievement of gross motor milestones such as ability to sit upright, walk, climb stairs; 6-minute walk test; CHOP INTEND; Egen Klassifikation; North Star Ambulatory Assessment; Hammersmith Functional Motor Scale-Expanded; Patient-Reported Outcomes Measurement Information System)
    - respiratory status (eg, pulmonary function tests, use of ventilatory support, occurrence of respiratory infection)
    - growth/nutrition (eg, growth in reference to WHO standards, requirement for supplemental feeding/feeding tube)
    • PK of dC/dT (Cmax, Tmax, AUC, t1/2)
    • Biomarkers (plasma), including those which may be related to TK2 disease and/or drug treatment (eg, creatine kinase)
    • Quality of life (eg, INQOL Questionnaire)
    • Characterize the health care utilization of TK2 patients, including hospitalization, surgeries, and use of health care resources (respiratory care, nursing care, physical therapy, etc.)
    • Los criterios de valoración de la eficacia pueden incluir los siguientes:
    - evaluaciones de la función motora (p. ej., logro de hitos de motricidad gruesa, tales como la capacidad para sentarse en posición vertical, caminar, subir escaleras; prueba de la marcha de 6 minutos; CHOP INTEND; Egen Klassifikation; Evaluación ambulatoria North Star [North Star Ambulatory Assessment]; Escala motora funcional de Hammersmith ampliada [Hammersmith Functional Scale-Expanded]; sistema de información de medición de los resultados notificados por el paciente)
    - estado respiratorio (p. ej., pruebas de función pulmonar, uso de asistencia ventilatoria, aparición de infección respiratoria)
    - crecimiento/nutrición (p. ej., crecimiento en referencia a los estándares de la OMS, requisito de alimentación complementaria/sonda de alimentación)
    • FC de dC/dT (Cmáx., Tmáx., ABC, t1/2)
    • Biomarcadores (plasma), incluidos los que puedan estar relacionados con la enfermedad de TK2 y/o el tratamiento farmacológico (p. ej., creatina cinasa)
    • Calidad de vida (p. ej., cuestionario INQOL)
    • Caracterizar la utilización de los recursos sanitarios de los pacientes TK2, como hospitalizaciones, intervenciones quirúrgicas y uso de recursos sanitarios (atención respiratoria, cuidados de enfermería, fisioterapia, etc.)
    E.5.2.1Timepoint(s) of evaluation of this end point
    See Schedule of Assessments
    Ver calendario de evaluaciones
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 23
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 14
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    infants and children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 21
    F.4.2.2In the whole clinical trial 38
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Following completion of the study patients will continue treatment under an Expanded Access program or be moved onto commercial drug.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-07-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-15
    P. End of Trial
    P.End of Trial StatusOngoing
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