BKM120 for Patients With PI3K-activated Tumors

Phase 2

Completed

Conditions: PI3K Pathway Activated Tumors

Interventions: Drug: BKM120

Registration Number: NCT01833169

Lead Sponsor: Novartis Pharmaceuticals

Brief Summary: The purpose of this signal seeking study was is to determine whether treatment with BKM120 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 146

Inclusion Criteria

Patient had a confirmed diagnosis of a solid tumor or hematological malignancy with the exception of endometrial cancer, glioblastoma, nonsmall cell lung cancer, prostate cancer or breast cancer.
Patient's tumor was evaluated and pre-identified to have activation of the PI3K pathway, at a CLIA certified laboratory
Patient must have received at least one prior treatment for recurrent metastatic and /or locally advanced disease and for whom no standard therapy options was anticipated to result in a durable remission.
Patient must have had progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines
Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

Exclusion Criteria

Patient had received previous treatment with BKM120 Patient had symptomatic CNS metastases Patient had mood disorder as outlined in Section 5 Patient had received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BKM120	BKM120	BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28- day cycle

Primary Outcome Measures

Name	Time	Method
Participant Clinical Benefit Response Rate	Week 16	Clinical benefit rate for patients with solid tumors will be assessed using RECIST 1.1 and will include responses of Complete Response (CR) or Partial Response (PR) or Stable Disease (SD) at \>=16 weeks. For hematologic tumors other appropriate hematological response criteria was applied. Response criteria: CR=Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm., PR=At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters, SD=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study, PD= At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline

Secondary Outcome Measures

Name	Time	Method
Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages	baseline up to 24 months	Progression free survival (PFS) is defined as the time from the date of first dose to the date of first documented disease progression or relapse or death due to any cause.
Overall Survival - Number of Participants With an Event	Every 8 Weeks until death, assessed up to 24 months	Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause. If a patient is not known to have died, survival time will be censored at the date of the last contact
Overall Survival (OS)- Kaplan-Meier Estimates of OS Timing in Months	baseline up to 24 months	Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause. If a patient is not known to have died, survival time will be censored at the date of the last contact
Overall Response of Partial Response (PR) or Greater. PR=at Least a 30% Decrease in the Sum of Diameters of Target Lesions, Taking as Reference the Baseline Sum Diameters	baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months	Overall Response (OR) of Partial Response (PR) or greater is based on local investigator assessment. For patients with solid tumors, the assessment criteria will be RECIST 1.1 and will include responses of CR and/or PR. For hematologic tumors other appropriate hematological response criteria apply
Progression-Free Survival - Number of Participants With an Event	Every 8 Weeks until death, assessed up to 24 months	Progression free survival (PFS) is defined as the time from the date of first dose to the date of first documented disease progression or relapse or death due to any cause
Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months	baseline up to 24 months	Progression free survival (PFS) is defined as the time from the date of first dose to the date of first documented disease progression or relapse or death due to any cause
Overall Survival (OS)- Kaplan-Meier Estimates of OS Rate in Percentages	baseline up to 30 months	Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause. If a patient is not known to have died, survival time will be censored at the date of the last contact

Trial Locations

Locations (59): Arizona Oncology Associates HOPE Division
🇺🇸
Phoenix, Arizona, United States
Arizona Oncology Associates PC- HAL
🇺🇸
Sedona, Arizona, United States
Highlands Oncology Group
🇺🇸
Fayetteville, Arkansas, United States
Sarcoma Oncology Center
🇺🇸
Santa Monica, California, United States
Rocky Mountain Cancer Centers RMCC - Aurora
🇺🇸
Greenwood Village, Colorado, United States
Whittingham Cancer Center Norwalk Hospital
🇺🇸
Norwalk, Connecticut, United States
Eastern Connecticut Hematology & Oncology Associates The Norwich Cancer Center
🇺🇸
Norwich, Connecticut, United States
Florida Cancer Specialists Dept of Oncology (2)
🇺🇸
Fort Myers, Florida, United States
Florida Hospital Cancer Institute FL Hosp. Cancer Instit.
🇺🇸
Orlando, Florida, United States
Florida Cancer Specialists
🇺🇸
West Palm Beach, Florida, United States
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Arizona Oncology Associates HOPE Division
🇺🇸Phoenix, Arizona, United States