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    EudraCT Number:2020-002100-39
    Sponsor's Protocol Code Number:PTC743-MIT-001-EP
    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:2021-02-09
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2020-002100-39
    A.3Full title of the trial
    Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)
    Estudio de la eficacia y seguridad de Vatiquinona en el tratamiento de la epilepsia farmacorresistente en pacientes con enfermedad mitocondrial.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
    Un estudio para evaluar la eficacia y seguridad de Vatiquinona para el tratamiento de la epilepsia farmacorresistente en pacientes con enfermedad mitocondrial
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberPTC743-MIT-001-EP
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04378075
    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 SponsorPTC 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 supportPTC THERAPEUTICS, INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC THERAPEUTICS, INC.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 CORPORATE COURT
    B.5.3.2Town/ citySOUTH PLAINFIELD
    B.5.3.3Post codeNJ 07080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 866-282-5873
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVatiquinone
    D.3.2Product code PTC743
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNVATIQUINONE
    D.3.9.1CAS number 1213269-98-7
    D.3.9.2Current sponsor codePTC743
    D.3.9.3Other descriptive nameEPI743; alpha-tocotrienol quinone
    D.3.9.4EV Substance CodeSUB188275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Genetically determined mitochondrial disease and associated refractory epilepsy
    Enfermedad mitocondrial determinada genéticamente y asociada a la epilepsia farmacorresistente.
    E.1.1.1Medical condition in easily understood language
    Mitochondrial disease associated with refractory epilepsy
    Epilepsia farmacorresistente asociada a la enfermedad mitocondrial.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10077953
    E.1.2Term Refractory epilepsy
    E.1.2System Organ Class 100000004852
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10052637
    E.1.2Term Genetic mitochondrial abnormalities NEC
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the effect of vatiquinone (PTC743) on reduction in observable motor seizure frequency in subjects with genetically confirmed mitochondrial disease, as assessed by a seizure diary.
    Demostrar el efecto de la vatiquinona (PTC743) sobre la reducción de la frecuencia de crisis motoras observables en sujetos con enfermedad mitocondrial confirmada genéticamente, en su valoración mediante un diario de crisis
    E.2.2Secondary objectives of the trial
    ● To demonstrate the effects of vatiquinone on seizure-related emergency room visits and hospitalizations
    ● To demonstrate the effects of vatiquinone on occurrence of status epilepticus
    ● To demonstrate the effects of vatiquinone in monthly total seizure count
    ● To demonstrate the effects of vatiquinone in responder rate in motor and non-motor seizures
    ● To demonstrate the effects of vatiquinone on number of rescue antiepileptic medications used
    ● To demonstrate the effects of vatiquinone on health-related quality of life (using the CarerQoL-7D questionnaire)
    ● To demonstrate the effects of vatiquinone on occurrence of seizure clusters
    ● To demonstrate the safety of vatiquinone as assessed by drug-related SAEs, drug-related adverse events (AEs), and dose modifications
    - Evaluar los efectos de la vatiquinona sobre las visitas a Urgencias y las hospitalizaciones por crisis
    - Evaluar los efectos de la vatiquinona sobre la presentación de status epilepticus
    - Evaluar los efectos de la vatiquinona sobre el número mensual total de crisis
    - Evaluar los efectos de la vatiquinona sobre la tasa de respondedores en cuanto a crisis motoras y no motoras
    - Evaluar los efectos de la vatiquinona sobre el número de medicamentos antiepilépticos de rescate utilizados
    - Evaluar los efectos de la vatiquinona sobre la calidad de vida relacionada con la salud (utilizando el cuestionario CarerQoL-7D)
    - Evaluar los efectos de la vatiquinona sobre la producción de crisis en racimo
    - Evaluar la seguridad de la vatiquinona, en su evaluación mediante los acontecimientos adversos graves relacionados con el fármaco (SAEs), los acontecimientos adversos relacionados con el fármaco (AEs) y las modificaciones de la dosis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Signed informed consent form.
    •Genetic confirmation of inherited mitochondrial disease (secondary to either nuclear or mitochondrial DNA mutation) with associated epilepsy phenotype

    •Despite ongoing treatment with at least 2 antiepileptic drugs:
    ◦have ≥6 observed motor seizures occurring during the 28 days prior to the baseline visit (Day 0).
    ◦have ≥2 observed motor seizures in the first 14 days and ≥2 in the second 14 days of the Run-in period (Day -14).
    ◦do not have a consecutive 20-day seizure free period.
    ◦have at least 80% of seizure diary data.
    No changes to the AED regimen will be allowed during the first 24-week period.

    •Documented medical history of epilepsy symptoms associated with mitochondrial disease for at least 6 months prior to screening.
    •Consent to abstain from non-approved therapies for 30 days prior to the baseline visit (Day 0) and for the duration of the study.
    •Stable dose regimen of antiepileptic therapies 30 days prior to the baseline visit (Day 0).
    •Stable regimen of dietary supplements 30 days prior and, if on a ketogenic diet, stable ketogenic diet 90 days prior to the baseline visit (Day 0) and for duration of the study.
    •Electroencephalogram (EEG) at screening or historical EEG (taken within the last 30 days) for diagnostic confirmation of epilepsy.
    - Formulario de consentimiento informado firmado
    - Confirmación genética de enfermedad mitocondrial hereditaria (secundaria a mutación del ácido desoxirribonucleico nuclear o mitocondrial), con fenotipo de epilepsia asociada

    - A pesar del tratamiento en curso con como mínimo 2 fármacos antiepilépticos:
    -ha presentado >= 6 crisis motoras observadas durante los 28 días anteriores a la visita basal (Dia 0)
    -ha presentado>=2 crisis motoras observadas en los primeros 14 días y >=2 en los segundos 14 días del periodo de preinclusión
    - no ha tenido un periodo de 20 días consecutivos sin crisis, y
    - su diario de crisis contiene como mínimo un 80% de los datos
    - No se permitirán cambios en el régimen de fármacos antiepilépticos durante el primer periodo de 24 semanas.
    - Antecedentes médicos documentados de epilepsia asociada a enfermedad mitocondrial durante como mínimo los 6 meses anteriores a la selección
    - Conformidad en no seguir tratamientos no aprobados durante los 30 días anteriores a la visita basal (día 0) y durante el estudio
    - Régimen estable de dosis de los medicamentos antiepilépticos durante los 30 días anteriores a la visita basal (día 0)
    - Régimen estable de suplementos dietéticos durante los 30 días anteriores y, en caso de dieta cetógena, esta se ha mantenido estable durante los 90 días anteriores a la visita basal y se mantendrá durante el estudio
    - Electroencefalograma (EEG) de la selección o de la historia clínica (tomada en los últimos 30 días) que confirma el diagnóstico de epilepsia
    E.4Principal exclusion criteria
    •Allergy to vatiquinone or sesame oil.
    •Aspartate transaminase (AST) or alanine transaminase (ALT) ≥2 × upper level of normal (ULN) at time of screening.
    •International normalized ratio (INR) ≥1.5 × ULN at time of screening.
    •Serum creatinine ≥1.5 × ULN at time of screening.
    •Participation in another interventional clinical trial 60 days prior to randomization or for the duration of this clinical trial
    •Previously received vatiquinone.
    •Concomitant treatment with drug(s) that have not received regulatory agency approval for the treatment of mitochondrial diseases.
    •Ongoing treatment with cytochrome P450 (CYP) inhibitors such as itraconazole or CYP inducers such as rifampin. Treatment with these agents must be completed at least 4 weeks prior to enrollment.
    •Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods. Females of childbearing potential must have a negative pregnancy test at screening and during the baseline visit (Day 0).
    - Alergia a la vatiquinona o al aceite de sésamo
    - Aspartato transaminasa (AST) o alanina transaminasa (ALR) >=2× límite superior de la normalidad (ULN) en la selección
    - Cociente internacional normalizado >=1,5×ULN en la selección
    - Creatinina sérica >=1,5×ULN en la selección
    - Participación en otro ensayo clínico intervencional en el plazo de los 60 días anteriores a la aleatorización o durante este ensayo clínico
    - Tratamiento previo con vatiquinona
    - Tratamiento concomitante con fármaco(s) no aprobado(s) por las autoridades sanitarias para el tratamiento de enfermedades de origen mitocondrial
    - Tratamiento en curso con inhibidores del citocromo P450 (CYP), como itraconazol, o inductores de CYP, como rifampicina. El tratamiento con estos agentes deberá haber finalizado como mínimo 4 semanas antes de la entrada en este estudio.
    - Mujeres embarazadas o en lactancia natural o sujetos sexualmente activos que no desean seguir métodos anticonceptivos adecuados. Las mujeres potencialmente fértiles deben arrojar una prueba de embarazo negativa en la
    selección y durante la visita basal (Dia 0)
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from Baseline to Week 24 in the Number of Observable Motor Seizures per 28 Days during the placebo-controlled phase
    Cambio porcentual desde la basal a la semana 24 en la frecuencia de crisis motoras observables por 28 días durante la fase controlada con placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 0, Week 24
    Dia 0, semana 24
    E.5.2Secondary end point(s)
    1.Number of Disease-Related Hospital Days
    2.Number of Participants with Occurrences or Recurrence of Status Epilepticus
    3.Number of Participants with Disease-Related In-Patient Hospitalizations or Emergency Room Visits
    4.Number of Disease-Related In-Patient Hospital Admissions or Emergency Room Visits
    5.Change from Baseline to Week 72 in Total Seizure Frequency (All Types) per 28 Days
    6.Percentage of Participants with ≥25%, ≥50%, ≥75%, and 100% Reduction in Motor Seizures
    7.Percentage of Participants with ≥25%, ≥50%, ≥75%, and 100% Reduction in Total Seizures (All Types)
    8.Number of Participants Who Require Rescue Seizure Medication
    9.Health-Related Quality of Life as Measured by the Care-Related Quality of Life of Informal Caregivers (CarerQoL-7D) Questionnaire
    10.Number of Participants with Seizure Clusters
    1. Número de días de hospitalización por la enfermedad
    2. Número de participantes con presentación o recidiva de status epilepticu
    3. Número y porcentaje de sujetos con hospitalizaciones/visitas a Urgencias relacionadas con la enfermedad
    4. Número de hospitalizaciones/visitas a Urgencias relacionadas con la enfermedad
    5. Cambio porcentual desde la basal a la semana 72 en la frecuencia total de crisis de todos los tipos por 28 días
    6. Porcentaje de sujetos con una reducción de las crisis motoras >=25%, >= 50%, >=75% y >=100%
    7. Porcentaje de sujetos con una reducción de todo tipo de crisis >=25%, >=50%, >=75% y >=100%
    8. Número de medicamentos de rescate para las crisis
    9. Calidad de vida relacionada con la salud, medida mediante el cuestionario CarerQoL-7D
    10. Número de crisis en racimo
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 24 and up to Week 72
    2. Week 24 and up to Week 72
    3. Week 24 and up to Week 72
    4. Week 24 and up to Week 72
    5. Day 0, Week 24, Week 72
    6. Week 24 and up to Week 72
    7. Week 24 and up to Week 72
    8. Week 24 and up to Week 72
    9. Week 24 and up to Week 72
    10. Week 24 and up to Week 72
    1. Semana 24 y hasta la semana 72
    2. Semana 24 y hasta la semana 72
    3. Semana 24 y hasta la semana 72
    4. Semana 24 y hasta la semana 72
    5. Día 0, semana 24, semana 72
    6. Semana 24 y hasta la semana 72
    7. Semana 24 y hasta la semana 72
    8. Semana 24 y hasta la semana 72
    9. Semana 24 y hasta la semana 72
    10. Semana 24 y hasta la semana 72
    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 No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    Ultima visita del último sujeto
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    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: 80
    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) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 40
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 80
    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)
    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 Decision2021-07-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-09
    P. End of Trial
    P.End of Trial StatusOngoing
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