E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult patient with metastatic or unresectable malignant perivascular epithelioid cell tumors (PEComa) |
Pacientes de 18 años de edad o mayores con PEComas malignos progresivos en estadio avanzado/irresecables o metastásicos. |
|
E.1.1.1 | Medical condition in easily understood language |
Adult patient with PEComa |
Pacientes de 18 años de edad o mayores con PEComa. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10039491 |
E.1.2 | Term | Sarcoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 determine the efficacy of BEZ235 in PEComa patients |
Determinar la eficacia de BEz235 en pacientes con PEComa |
|
E.2.2 | Secondary objectives of the trial |
To determine the PFS rate at 32 weeks in the included population To assess the duration of response among responders To evaluate time to response To evaluate the time to progression To assess the overall survival To evaluate safety and tolerability of BEZ235 |
Determinar la SLP a 32 semanas entre la población incluída Evaluar la duración de la respuesta entre los respondedores Evaluar el tiempo de respuesta Evaluar el tiempo hasta progresión Evaluar la supervivencia Evaluar la seguridad y tolerabilidad de BEZ235 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Histologically confirmed diagnosis of malignant PEComa (included epithelioid angiomyolipoma (AML)) in adult patients Unresectable/advanced and/or metatstatic and documented progressive measurable disease Treated with 1 or 2 prior lines of treatment |
Diagnóstico histológico confirmado de PEComa maligno (angiomiolipoma epitelioide (AML) incluido) en pacientes adultos Enfermedad avanzada/irresecable o metastásica medible progresiva documentada Pacientes tratados con 1 o 2 líneas de tratamiento previas. |
|
E.4 | Principal exclusion criteria |
Disease exclusions : lymphangioleiomyomatosis (LAM) exclusively, ative uncontrolled or symptomatic CNS metastases, concurrent malignancy or malignancy in last 3 years Concurrent severe and/or uncontrolled medical conditions (for details see protocol) Other protocol-defined inclusion/exclusion criteria may apply |
Exclusiones por enfermedada: limfagioleiomiomatosis (LAM) exclusivamente, metástasis activas no controladas o sintomáticas del SNC, Cáncer concurrente y/o condiciones médicas no controladas (consultar protocolo para más detalles) Pueden aplicar otros criterios de inclusión/exclusión definidos en el protocolo. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Objective Response Rate (best response on study) according to RECIST 1.1 criteria |
Determinar la eficacia de BEZ235 en la tasa de respuesta objetiva (mejor respuesta en estudio) según los criterios RECIST 1.1. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
32 weeks after the last enrolled patient |
32 semanas después del último paciente incluido |
|
E.5.2 | Secondary end point(s) |
PFS rate at 32 weeks of follow-up Duration of response using with RECIST 1.1 criteria Time to response Time to progression Overall survival Frequency and severity of adverse events and laboratory values abnormalities Other safety data as considered appropriate |
Determinar la tasa de supervivencia libre de progresión a las 32 semanas en la población incluida. Evaluar la duración de la respuesta entre los respondedores. Evaluar el tiempo hasta la respuesta. Evaluar el tiempo hasta la progresión. Evaluar la supervivencia global. Evaluar la seguridad y tolerabilidad de BEZ235. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
32 weeks after the last enrolled patient |
32 semanas después del último paciente incluido |
|
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 | Yes |
E.6.11 | Pharmacogenomic | Yes |
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 | 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.8.3 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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 | 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
|
1 year after the last patient has been included or when all patients die (whichever comes first) or when the study is terminated early |
El estudio finalizará 1 año después de que se haya incluido el ultimo paciente o cuando todos los pacientes hayan fallecido (aquello que ocurra primero) o cuando el estudio finalice de forma prematura. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 36 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 36 |
E.8.9.2 | In all countries concerned by the trial days | 0 |