E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Refractory seizures associated with tuberous sclerosis complex |
Crisis epilépticas refractarias asociadas al complejo de esclerosis tuberosa (CET). |
|
E.1.1.1 | Medical condition in easily understood language |
Refractory seizures associated with tuberous sclerosis complex |
Crisis epilépticas refractarias asociadas al complejo de esclerosis tuberosa (CET). |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10045138 |
E.1.2 | Term | Tuberous sclerosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 evaluate the long term safety data with everolimus treatment i.e adverse events (AEs) and serious adverse events (SAEs). |
Evaluar los datos de seguridad a largo plazo con el tratamiento con everolimus, es decir, acontecimientos adversos (AAs) y acontecimientos adversos graves (AAGs). |
|
E.2.2 | Secondary objectives of the trial |
To evaluate clinical benefit as assessed by the Investigator |
Evaluar el beneficio clínico, determinado por el investigador. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient is currently enrolled in the Novartis-sponsored study CRAD001M2304 receiving everolimus and has fulfilled all its requirements. 2. Patient is currently benefiting from treatment with everolimus, as determined by the Investigator. 3. Patient has demonstrated compliance, as assessed by the Investigator, with the parent study protocol requirements. 4. Patient is willing and able to comply with scheduled visits and treatment plans. 5. Written informed consent/adolescent assent obtained prior to enrolling into the roll-over study. |
1. Pacientes que estén participando actualmente en el estudio patrocinado por Novartis, CRAD001M2304, que reciban everolimus y que hayan cumplido todos sus requisitos. 2. Pacientes que se estén beneficiando actualmente del tratamiento con everolimus, determinado por el investigador 3. Pacientes que hayan cumplido con los requisitos del protocolo del estudio principal, determinado por el investigador. 4. Pacientes que estén dispuestos a y puedan cumplir con los planes de tratamiento y visitas programadas. 5. Consentimiento informado/asentimiento pediátrico por escrito obtenido antes de la inclusión en el estudio de continuación. |
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E.4 | Principal exclusion criteria |
1. Patient has been permanently discontinued from everolimus study treatment in CRAD001M2304. 2. Everolimus is approved for patients with TSC with refractory seizures 3. Patients who are receiving everolimus in combination with unapproved or experimental treatments for seizure control Anti-epileptic drug (AEDs) are allowed for the purpose of seizure control). |
1. Pacientes a los que se les haya retirado permanentemente el tratamiento del estudio, everolimus, en el estudio CRAD001M2304. 2. Everolimus esté aprobado para pacientes con CET y crisis epilépticas refractarias y este reembolsado en el país local. 3. Pacientes que estén recibiendo everolimus en combinación con tratamientos experimentales o no aprobados para el control de las crisis epilépticas (los fármacos antiepilépticos (AEDs) están permitidos para controlar las crisis epilépticas). |
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E.5 End points |
E.5.1 | Primary end point(s) |
Frequency and severity of AEs/SAEs |
Frecuencia y gravedad de los AAs/AAGs |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Continuous monitoring and reporting every 3 months (during scheduled visits) |
Monitorización continua e informe cada 3 meses (durante las visitas programadas) |
|
E.5.2 | Secondary end point(s) |
Proportion of patients with clinical benefit as assessed by the Investigator at scheduled visits |
Proporción de pacientes con beneficio clínico determinado por el investigador durante las visitas programadas. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Continuous monitoring and reporting every 3 months (during scheduled visits) |
Monitorización contínua e informe cada 3 meses (durante las visitas programadas) |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Roll-over study (after completion of Study CRAD001M2304 (EXIST-3)) |
Estudio de continuación (una vez han completado el estudio CRAD001M2304 (EXIST-3)) |
|
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 | Yes |
E.8.1.7.1 | Other trial design description |
Estudio de continuación. |
Roll-over study |
|
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 29 |
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 |
Australia |
Belgium |
Canada |
Colombia |
Denmark |
France |
Greece |
Hungary |
Japan |
Korea, Republic of |
Netherlands |
Poland |
Russian Federation |
Spain |
Taiwan |
Thailand |
Turkey |
United Kingdom |
United States |
|
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
|
LPLV |
Último paciente, última visita |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 10 |
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 | 10 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |