E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with CPVT and ICD |
Pazienti con CPVT e ICD |
|
E.1.1.1 | Medical condition in easily understood language |
Patients with catecholaminergic ventricular tachycardia and implantable defibrillator |
Pazienti con tachicardia ventricolare catecolaminergica e defibrillatore impiantabile |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028159 |
E.1.2 | Term | Multiform ventricular tachycardia |
E.1.2 | System Organ Class | 10007541 - Cardiac disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy of flecainide in reducing cardiac events in CPVT |
Valutare l'efficacia della Flecainide nel ridurre gli eventi cardiaci nella CPVT. |
|
E.2.2 | Secondary objectives of the trial |
- To assess the efficacy of flecainide in reducing ventricular ectopy and VT during exercise treadmill testing in CPVT - To correlate ventricular ectopy and VT during exercise treadmill testing with cardiac events in CPVT |
- valutare l'efficacia della Flecainide nel ridurre le extrasistoli ventricolari e le TV durante test da sforzo nella CPVT - correlare l’extrasistolia ventricolare e la TV durante il test da sforzo con gli eventi cardiaci nella CPVT. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Clinical diagnosis of CPVT, based on: reproducible polymorphic or bidirectional ventricular tachycardia with exercise or Ventricular ectopy on exercise test with RYR2 or CASQ2 mutation - Functioning ICD in place - On stable dose of standard therapy defined as the maximal tolerated dose of beta-blocker and may include a calcium channel blocker (Patients on flecainide or mexiletine are also eligible for enrollment after a 1 week “washout” period during which flecainide or mexiletine is discontinued, and standard therapy alone is used). |
- Diagnosi clinica di CPVT, sulla base di: tachicardia ventricolare polimorfica bidirezionale riproducibile con l'esercizio o extrasistolia ventricolare durante test da sforzo con mutazione RYR2 o CASQ2 - ICD funzionante - In terapia standard con dosaggio stabile definita come la dose massima tollerata di beta-bloccante e può includere un bloccante del canale del calcio (I pazienti in terapia con Flecainide o mexiletina sono ammissibili per l’arruolamento dopo una settimana di ''washout'', durante il quale viene interrotta la flecainide o la mexiletina, e viene utilizzata solo la terapia standard). |
|
E.4 | Principal exclusion criteria |
• Females who are pregnant or plan to be pregnant during the study period • Children < 5 years of age • Patients unable to perform treadmill exercise • Patients with significant structural heart disease • Patients with features consistent with Andersen-Tawil syndrome • Patients with known hypersensitivity to flecainide • Patients on amiodarone • Patients not expected to comply with follow-up |
• Le donne incinte o che hanno programmato una gravidanza durante il periodo di studio • Bambini <5 anni di età • I pazienti non in grado di eseguire esercizio al treadmill • I pazienti con significativa malattia cardiaca strutturale • I pazienti con caratteristiche tipiche della sindrome di Andersen-Tawil • I pazienti con nota ipersensibilità alla flecainide • I pazienti in amiodarone • I pazienti che non sono in grado di rispettare il follow-up |
|
E.5 End points |
E.5.1 | Primary end point(s) |
VT treated by ICD or death |
tachicardie ventricolari (TV) trattate con ICD oppure morte |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
3, 6, 12, 18 months after treatment |
3, 6, 12, 18 mesi dalla somministrazione |
|
E.5.2 | Secondary end point(s) |
- VT leading to syncope but not treated by ICD; symptomatic VT not treated by ICD - exercise duration; peak heart rate during exercise test |
- TV che provocano sincope ma non trattate con ICD; TV sintomatiche non trattate con ICD - durata dello sforzo, frequenza cardiaca massima durante test da sforzo. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
3, 6, 12, 18 months after treatment |
3, 6, 12, 18 mesi dalla somministrazione |
|
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 | No |
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 | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | No |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |