E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
E.1.1.1 | Medical condition in easily understood language |
Diffuse large B-cell lymphoma, Mantle cell lymphoma and Follicular lymphoma |
|
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 | 16.1 |
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 | 16.1 |
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) |
|
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 |
|
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.
Other protocol defined criteria may apply |
|
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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
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 |
|
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.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
up to 2 years when all cohorts are completed |
|
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 | 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 | 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 | 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 |
Belgium |
France |
Italy |
Brazil |
Germany |
Korea, Republic of |
Spain |
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 |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |