E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Inherited mitochondrial disease |
Malattia mitocondriale ereditaria |
|
E.1.1.1 | Medical condition in easily understood language |
Inherited mitochondrial disease |
Malattia mitocondriale ereditaria |
|
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 |
|
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 |
Valutare la sicurezza di vatichinone in soggetti affetti da malattia mitocondriale ereditaria con precedente esposizione a vatichinone in uno studio clinico o piano di trattamento sponsorizzato da PTC/BioElectron (in precedenza Edison). |
|
E.2.2 | Secondary objectives of the trial |
|
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. |
1. Soggetti con malattia mitocondriale ereditaria, tra cui sindrome di Leigh, sindrome di Alpers, encefalomiopatia mitocondriale, acidosi lattica ed episodi simili a ictus (MELAS), epilessia mioclonica con fibre rosse raggrinzite (MERRF), ipoplasia pontocerebellare di tipo 6 (PCH6) o altra malattia mitocondriale che hanno partecipato a un precedente studio clinico o piano di trattamento con vatichinone. 2. Le donne in età fertile, secondo la definizione del CTFG 2020, devono presentare un test di gravidanza negativo allo screening/basale e acconsentire all’astinenza o all’uso di almeno uno dei seguenti metodi contraccettivi altamente efficaci (con un tasso di insuccesso < 1% all’anno, se utilizzati in modo costante e corretto). L’uso di contraccezione altamente efficace o la pratica dell’astinenza deve estendersi per tutta la durata dello studio e fino a 50 giorni dopo l’ultima dose di farmaco dello studio: • contraccezione ormonale combinata (a base di estrogeni e progestinici) associata a inibizione dell’ovulazione: - orale - intravaginale - transdermica • contraccezione ormonale a base di soli progestinici associata a inibizione dell’ovulazione: - orale - iniettabile - impiantabile • dispositivo intrauterino • sistema di rilascio ormonale intrauterino • partner vasectomizzato con azoospermia confermata Tutte le donne saranno considerate in età fertile, a meno che non siano in post-menopausa (in amenorrea da almeno 12 mesi consecutivi nella fascia di età appropriata e in assenza di altra causa nota o sospetta) o si siano sottoposte a sterilizzazione chirurgica (ad esempio, tramite legatura delle tube bilaterale, isterectomia, ooforectomia bilaterale). 3. Uomini fertili, secondo la definizione del CTFG 2020, che siano sessualmente attivi con donne in età fertile e che non si siano sottoposti a vasectomia, devono accettare di utilizzare un metodo contraccettivo barriera durante lo studio e per un massimo di 50 giorni dopo l’ultima dose di farmaco dello studio. |
|
E.4 | Principal exclusion criteria |
1. Current participation in any other interventional study. 2. Pregnancy or breast feeding. |
1. Attuale partecipazione a qualsiasi altro studio interventistico 2. Gravidanza o allattamento |
|
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) |
EA/SAE, ECG, segni vitali e dati di laboratorio (dati di ematologia, coagulazione, biochimica e urine). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
throughout study |
Per tutta la durata dello studio |
|
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 |
open label extension |
estensione in aperto |
|
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
nessun comparatore |
no comparator |
|
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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 | Information not present in EudraCT |
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 |
Australia |
Canada |
United States |
France |
Spain |
Italy |
United Kingdom |
|
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
|
The end of the study per subject disposition will be the date of the last study visit for the last subject in the study. |
La fine dello studio per disposizione del soggetto sarà la data dell'ultima visita di studio per l'ultimo soggetto dello studio. |
|
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 | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
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 |