Clinical trial NCT01693614: Safety and Efficacy of BKM120 in Relapsed and Refractory NHL
Official title: An Open-label Phase II Study of BKM120 in Subjects With Relapsed and Refractory Diffuse Large B-cell Lymphoma, Mantle Cell Lymphoma and Follicular Lymphoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Diffuse Large B-cell Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma
Keywords: Diffuse large B-cell lymphoma, Mantle cell lymphoma, Follicular lymphoma, PI3K inhibitor, Non-Hodgkin lymphoma, NHL
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-09-26
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a phase II study evaluating the safety, tolerability and efficacy of BKM120 in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL) or follicular lymphoma (FL).


Eligibility criteria: Inclusion Criteria:

1. Patient had a histologically confirmed diagnosis of mantle cell lymphoma, follicular lymphoma, or diffuse large B cell lymphoma.
2. Patient had relapsed or refractory disease and received at least one prior therapy.
3. Patient with diffuse large B cell lymphoma had received or was ineligible for autologous or allogeneic stem cell transplant.
4. Patient had at least one measurable nodal lesion (≥2 cm) according to Cheson criteria (Cheson 2007). In case where the patient had no measurable nodal lesions ≥ 2 cm in the long axis at baseline, then the patient must have had at least one measurable extra-nodal lesion.
5. Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
6. Patient had adequate bone marrow and organ function.

Exclusion Criteria:

1. Patient had received previous treatment with PI3K inhibitors
2. Patient had evidence of graft versus host disease (GVHD).
3. Patient had active or history of central nervous system (CNS) disease.
4. Patient had a concurrent malignancy or had 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 had a score ≥ 12 on the PHQ-9 questionnaire.
6. Patient had a GAD-7 mood scale score ≥ 15.
7. Pregnant or nursing women
8. Patient who did not use highly effective contraception methods to avoid becoming pregnant or conceiving offspring.

Primary outcomes:
measure: Overall Response Rate (ORR) and Disease Control Rate (DCR) Per Investigator at 6 Months (FAS)
timeFrame: Baseline up to 6 months
Secondary outcomes:
measure: Progression- Free Survival (PFS) Based on Investigator Assessment (FAS)
timeFrame: Baseline up to approximately 44 months
measure: Duration of Response for Diffuse Large B-cell Lymphoma (DLBCL), and Follicular Lymphoma (FL) Cohorts (FAS)
timeFrame: Baseline up to approximately 18 months
measure: Overall Survival (OS) - Percentage of Participants With OS Events (FAS)
timeFrame: Baseline up to approximately 44 months
measure: Percentage of Participants - Overall Survival- Kaplan Meier Estimates (FAS)
timeFrame: Baseline up to approximately 18 months
measure: Overall Survival - Median (FAS)
timeFrame: Baseline up approximately 44 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Omaha
country: United States
facility: University of Nebraska Medical Center Univ Nebraska
state: Nebraska
city: New York
country: United States
facility: Memorial Sloan Kettering Dept of Onc.
state: New York
city: Charleston
country: United States
facility: Medical University of South Carolina -Hollings Cancer Center Medical Univ of South Carolina
state: South Carolina
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center Dept.ofMDAndersonCancerCtr(3)
state: Texas
city: Brugge
country: Belgium
facility: Novartis Investigative Site
city: Yvoir
country: Belgium
facility: Novartis Investigative Site
city: Marseille
country: France
facility: Novartis Investigative Site
city: Pierre-Benite Cedex
country: France
facility: Novartis Investigative Site
city: Rennes
country: France
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Wurzburg
country: Germany
facility: Novartis Investigative Site
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Gyeonggi-do
country: Korea, Republic of
facility: Novartis Investigative Site
state: Korea
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
state: Korea
city: Salamanca
country: Spain
facility: Novartis Investigative Site
state: Castilla Y Leon
city: Hospitalet de LLobregat
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: Samsun
country: Turkey
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Younes A, Salles G, Martinelli G, Bociek RG, Barrigon DC, Barca EG, Turgut M, Gerecitano J, Kong O, Pisal CB, Tavorath R, Kim WS. Pan-phosphatidylinositol 3-kinase inhibition with buparlisib in patients with relapsed or refractory non-Hodgkin lymphoma. Haematologica. 2017 Dec;102(12):2104-2112. doi: 10.3324/haematol.2017.169656. Epub 2017 Sep 29.