E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Dravet syndrome or Lennox-Gastaut syndrome |
Sindrome di Dravet o sindrome di Lennox-Gastaut |
|
E.1.1.1 | Medical condition in easily understood language |
Dravet syndrome or Lennox-Gastaut syndrome |
Sindrome di Dravet o sindrome di Lennox-Gastaut |
|
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 | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10048816 |
E.1.2 | Term | Lennox-Gastaut syndrome |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10073682 |
E.1.2 | Term | Dravet syndrome |
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 long-term safety and tolerability of ZX008 |
Valutare la sicurezza e la tollerabilità a lungo termine di ZX008 |
|
E.2.2 | Secondary objectives of the trial |
To assess the effect of ZX008 on the following effectiveness measures: - Investigator assessment of convulsive seizure response (<25%, >= 25%,>= 50%, >= 75%, or 100% [ie, seizure-free] improvement) - Clinical Global Impression – Improvement (CGI-I) rating, global and symptomatic, as assessed by the investigator - CGI-I rating, global and symptomatic, as assessed by the parent/caregiver |
Valutare l’effetto di ZX008 sulle seguenti misure di efficacia: - Valutazione dello sperimentatore della risposta alle crisi convulsive (miglioramento del <25%, >= 25%, >= 50%, >= 75%, o 100% [ovvero, libero da crisi convulsive]) - Valutazione dell’impressione clinica globale – Miglioramento (Clinical Global Impression– Improvement, CGI-I), globale e sintomatica, valutata dallo sperimentatore - Valutazione della CGI-I, globale e sintomatica, valutata dal genitore/dalla persona che assiste il soggetto |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Male or non pregnant, non lactating female • Satisfactory completion of a core study • Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008 • Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability |
- Soggetto di sesso maschile o di sesso femminile non in stato di gravidanza né in allattamento - Completamento soddisfacente dello studio core - Ha un raro disturbo convulsivo, come l'encefalopatia epilettica ed ha completato con successo altri studi clinici sponsorizzati da Zogenix con ZX008 - La persona che assiste il soggetto è disposta e in grado di rispettare la conformità alle procedure dello studio, al programma delle visite e alla contabilità del farmaco dello studio. |
|
E.4 | Principal exclusion criteria |
• Current cardiac valvulopathy or pulmonary hypertension that is clinically significant • Moderate or severe hepatic impairment • Receiving prohibited medication (please see protocol section 5.6.2), within 14 days of receiving ZX008 |
- Il soggetto presenta un’attuale valvulopatia o ipertensione polmonare clinicamente significativa - Insufficienza epatica moderata o grave - Somministrazione di farmaci vietati (si faccia riferimento alla sezione 5.6.2 del protocollo) entro 14 giorni dal trattamento con ZX008 |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The safety endpoints of the study are: • AEs • Laboratory safety (hematology, chemistry) • Vital signs (blood pressure, heart rate, temperature, and respiratory rate) • Physical examination • Neurological examination • Electrocardiogram (ECGs) • Doppler echocardiogram (ECHOs) • Body weight/height • Chest x-ray (subjects in France and Netherlands only) • Electroencephalogram (EEG) (in Italy only) |
Gli endpoint di sicurezza dello studio sono: • EA • Sicurezza di laboratorio (ematologia, ematochimica) • Parametri vitali (pressione sanguigna, frequenza cardiaca, temperatura corporea e frequenza respiratoria) • Esame obiettivo • Esame neurologico • Elettrocardiogramma (ECG) • Ecocardiogramma Doppler (ECHO) • Peso corporeo / altezza • Radiografia del torace (soggetti solo in Francia e Paesi Bassi) • Elettroencefalogramma (EEG) (solo in Italia) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 36 months |
Fino a 36 mesi |
|
E.5.2 | Secondary end point(s) |
The effectiveness endpoints of the study are: • CGI-I, global and symptomatic, as assessed by parent/caregiver • CGI-I, global and symptomatic, as assessed by investigator (or designee) • Percent improvement in seizure burden as assessed by the investigator (or designee) |
Gli endpoint di efficacia dello studio sono: • CGI-I, globale e sintomatica, valutata dal genitore/dalla persona che assiste il soggetto • CGI-I, globale e sintomatica, valutata dallo sperimentatore (o da un suo incaricato) • Percentuale di miglioramento del carico delle crisi convulsive, valutata dallo sperimentatore (o da un suo incaricato) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 36 months |
Fino a 36 mesi |
|
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 | No |
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 | 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) | 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 | No |
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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 43 |
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 |
Japan |
Mexico |
United States |
Belgium |
Denmark |
France |
Germany |
Italy |
Netherlands |
Poland |
Spain |
Sweden |
United Kingdom |
|
E.8.7 | Trial 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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 22 |