E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Inherited mitochondrial disease
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Maladie mitochondriale héréditaire
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E.1.1.1 | Medical condition in easily understood language |
Inherited mitochondrial disease |
Maladie mitochondriale héréditaire
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10052637 |
E.1.2 | Term | Genetic mitochondrial abnormalities NEC |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety of vatiquinone in subjects with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron-sponsored (previously Edison) clinical study or treatment plan
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Évaluer la sécurité de la vatiquinone chez des patients atteints d’une maladie mitochondriale héréditaire ayant déjà été exposés à la vatiquinone dans le cadre d’une étude clinique ou d’un plan de traitement financé(e) par PTC/BioElectron (précédemment Edison) |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects with inherited mitochondrial disease including Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan. 2. Women of childbearing potential, as defined in (CTFG 2020), must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least one of the following highly effective forms of contraception (with a failure rate of <1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 50 days after the last dose of study drug: • combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: − oral − intravaginal − transdermal • progestogen-only hormonal contraception associated with inhibition of ovulation: − oral − injectable − implantable • intrauterine device • intrauterine hormone-releasing system • vasectomized partner with confirmed azoospermia All females will be considered of childbearing potential unless they are postmenopausal (at least 12 months consecutive amenorrhea in the appropriate age group without other known or suspected cause) or have been sterilized surgically (eg, bilateral tubal ligation, hysterectomy, bilateral oophorectomy). 3. Fertile men, as defined in (CTFG 2020), who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug.
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1. Les patients doivent présenter une maladie mitochondriale héréditaire, y compris un syndrome de Leigh, un syndrome d’Alpers, une encéphalomyopathie mitochondriale, une acidose lactique et des épisodes déficitaires neurologiques (syndrome MELAS), une épilepsie myoclonique associée à la myopathie des fibres rouges en haillons (syndrome MERRF), une hypoplasie pontocérébelleuse de type 6 (PCH6) ou d’autres maladies mitochondriales, et avoir participé à une étude clinique ou à un plan de traitement antérieur sur la vatiquinone. 2. Les femmes en âge de procréer, telles que définies dans (CTFG 2020), doivent présenter un test de grossesse négatif lors de la sélection/l’inclusion et accepter de pratiquer l’abstinence ou d’utiliser au moins l’une des méthodes de contraception hautement efficaces suivantes (associées à un taux d’échec < 1 % par an lorsqu’elles sont utilisées de manière continue et correcte). L’utilisation de la méthode de contraception hautement efficace ou la pratique de l’abstinence doit être maintenue pendant toute la durée de l’étude et pendant 50 jours après la dernière dose de médicament à l’étude : • contraception hormonale combinée (contenant un œstrogène et un progestatif) associée à une inhibition de l’ovulation : − orale, − intravaginale, − transdermique ; • contraception hormonale contenant uniquement un progestatif associée à une inhibition de l’ovulation : − orale, − injectable, − implantable ; • dispositif intra-utérin (DIU) ; • dispositif intra-utérin hormonal ; • partenaire vasectomisé avec azoospermie confirmée. Toutes les femmes seront considérées comme étant en âge de procréer, sauf si elles sont ménopausées (au moins 12 mois consécutifs d’aménorrhée dans la tranche d’âge appropriée sans autre cause connue ou suspectée) ou si elles ont été stérilisées chirurgicalement (p. ex. : ligature bilatérale des trompes, hystérectomie, ovariectomie bilatérale). 3. Les hommes fertiles, tels que définis dans (CTFG 2020), qui sont sexuellement actifs avec des femmes en âge de procréer et qui n’ont pas subi de vasectomie, doivent accepter d’utiliser une méthode de contraception de type barrière pendant l’étude et pendant 50 jours après la dernière dose de médicament à l’étude. |
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E.4 | Principal exclusion criteria |
1. Current participation in any other interventional study. 2. Pregnancy or breast feeding.
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1. Participation actuelle à toute autre étude interventionnelle. 2. Grossesse ou allaitement.
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E.5 End points |
E.5.1 | Primary end point(s) |
Adverse events (AEs)/SAEs, ECGs, vital signs, and laboratory data (hematology, biochemistry, and urine data)
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Événements indésirables (EI)/EIG, ECG, signes vitaux et données biologiques (données hématologiques, biochimiques et urinaires)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
throughout study |
tout au long de l'étude |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Japan |
United States |
France |
Italy |
Poland |
Spain |
Sweden |
United Kingdom |
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E.8.7 | Trial 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
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The end of the study per subject disposition will be the date of the last study visit for the last subject in the study.
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La fin de l'étude par patient sera la date de la dernière visite du dernier patient de l'étude. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 4 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 16 |