E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Diffuse large B-cell lymphoma, Mantle cell lymphoma and Follicular lymphoma |
Linfoma B diffuso a grandi cellule, linfoma mantellare e linfoma follicolare |
|
E.1.1.1 | Medical condition in easily understood language |
Diffuse large B-cell lymphoma, Mantle cell lymphoma and Follicular lymphoma |
Linfoma B diffuso a grandi cellule, linfoma mantellare e linfoma follicolare |
|
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 | 10012822 |
E.1.2 | Term | Diffuse large B-cell lymphoma refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 | 10026801 |
E.1.2 | Term | Mantle cell lymphoma refractory |
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 BKM120 in patients with relapsed/refractory Non-Hodgkin Lymphoma in the three different histological subgroups (cohorts) |
Determinare l’efficacia di BKM120 in pazienti affetti da NHL recidivante/refrattario in tre diversi sottogruppi istologici. |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate the safety and tolerability in the three different histological subgroups; - To assess progression free survival in the three different histological subgroups; - To assess the duration of response in the three different histological subgroups; - To assess overall survival in the three different histological subgroups. |
- Valutare la sicurezza d’impiego e la tollerabilità nei tre diversi sottogruppi istologici; - Valutare la sopravvivenza libera da progressione (Progression Free Survival –PFS) nei tre diversi sottogruppi istologici; - Valutare la durata della risposta nei tre diversi sottogruppi istologici; - Valutare la sopravvivenza globale nei tre diversi sottogruppi istologici. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Patient has a histologically confirmed diagnosis of mantle cell lymphoma, follicular lymphoma, or diffuse large B cell lymphoma. 2. Patient has relapsed or refractory disease and received at least one prior therapy. 3. Patient with diffuse large B cell lymphoma has received or is ineligible for autologous or allogeneic stem cell transplant. 4. Patient has at least one measurable nodal lesion (≥2 cm) according to Cheson criteria (Cheson 2007). In case where the patient has no measurable nodal lesions ≥ 2 cm in the long axis at baseline, then the patient must have at least one measurable extra-nodal lesion. 5. Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2. 6. Patient has adequate bone marrow and organ function. |
1. Pazienti con diagnosi confermata istologicamente di linfoma mantellare, linfoma follicolare, o linfoma diffuso a grandi cellule B; 2. Pazienti con malattia recidivante o refrattaria che hanno ricevuto almeno una terapia precedente; 3. Pazienti con linfoma diffuso a grandi cellule B che hanno ricevuto o non sono eleggibili per un trapianto di cellule staminali autologo o allogenico. Questo criterio non si applica ai pazienti affetti da linfoma mantellare e linfoma follicolare; 4. Pazienti con almeno una lesione nodale misurabile (≥ 2 cm) secondo i criteri Cheson (Cheson 2007). Nel caso in cui il paziente non abbia lesioni nodali misurabili ≥ 2 cm nell’asse lungo al basale, il paziente deve avere almeno una lesione misurabile extra-nodale; 5. Pazienti con un performance status Eastern Cooperative Oncology Group (ECOG) ≤ 2; 6. Pazienti con adeguata funzionalità midollare e d’organo. |
|
E.4 | Principal exclusion criteria |
1. Patient has received previous treatment with PI3K. 2. Patient has evidence of graft versus host disease (GVHD). 3. Patient has active or history of central nervous system (CNS) disease. 4. Patient has a concurrent malignancy or has a malignancy within 3 years of study enrollment (with the exception of adequately treated basal or squamous cell carcinoma or non-melanomatous skin cancer). 5. Patient has a score ≥ 12 on the PHQ-9 questionnaire. 6. Patient has a GAD-7 mood scale score ≥ 15. 7. Pregnant or nursing women and who does not use highly effective contraception methods to avoid becoming pregnant or conciving offspring. Other protocol-defined inclusion/exclusion criteria may apply. |
1. Pazienti che hanno ricevuto un trattamento precedente con inibitori PI3K e/o mTOR; 2. Pazienti con evidenza di malattia del trapianto contro l’ospite (Graft Versus Host Disease – GVHD); 3. Pazienti con malattia attiva o anamnesi di malattia del sistema nervoso centrale (Central Nervous System – CNS); 4. Pazienti con patologie maligne concomitanti o con patologia maligna nei 3 anni precedenti l’arruolamento nello studio (ad eccezione del carcinoma basocellulare o del carcinoma squamocellulare o del carcinoma cutaneo non melanomatoso adeguatamente trattati)5. Pazienti con un punteggio ≥ 12 al questionario PHQ-9; 6. Pazienti con un punteggio ≥ 15 nella scala per la valutazione del tono dell’umore GAD-7 . |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Objective Response rate (ORR) |
Tasso di risposta obiettiva (Objective Response Rate - ORR) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to approx. 2 years when all cohorts are completed |
Fino a circa 2 anni una volta completate tutte le coorti |
|
E.5.2 | Secondary end point(s) |
- Frequency and severity of adverse events. Other safety data as considered appropriate; - PFS progression free survival; - Duration of response; - OS Overall survival. |
- Frequenza e severità degli eventi avversi. Altri dati di sicurezza d’impiego come considerato appropriato; - Sopravvivenza libera da progressione (Progression Free Survival - PFS); - Durata della risposta; - Sopravvivenza globale (Overall Survival - OS). |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 2 years when all cohorts are completed |
Fino a 2 anni una volta completate tutte le coorti |
|
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 | Yes |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 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 | 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 |
Brazil |
Korea, Republic of |
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
|
The End of the trial will be when all patients in each cohort have completed their 6 months assessment or discontinued early |
Lo studio si concluderà quando tutti i pazienti di ogni coorte avranno completato i 6 mesi di valutazione o interrotto prematuramente. |
|
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 | 23 |
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 | 23 |
E.8.9.2 | In all countries concerned by the trial days | 0 |