Phase 2
N=146
BKM120 for Patients With PI3K-activated Tumors
PI3K Pathway Activated Tumors
Bottom Line
View on ClinicalTrials.gov: NCT01833169 ↗Enrolled (actual)
146
Serious AEs
40.4%
Results posted
Dec 2017
Primary outcome: Primary: Participant Clinical Benefit Response Rate — 15.1 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BKM120 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Sep 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Participant Clinical Benefit Response Rate |
15.1 | — |
| SECONDARY 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 |
1.4 | — |
| SECONDARY Progression-Free Survival - Number of Participants With an Event |
112; 34 | — |
| SECONDARY Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months |
1.9 | — |
| SECONDARY Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages |
88.4; 43.8; 39.4; 20.4; 20.4; 14.1 | — |
| SECONDARY Overall Survival - Number of Participants With an Event |
110; 36 | — |
| SECONDARY Overall Survival (OS)- Kaplan-Meier Estimates of OS Timing in Months |
6.3 | — |
| SECONDARY Overall Survival (OS)- Kaplan-Meier Estimates of OS Rate in Percentages |
97.3; 83.4; 73.5; 61.5; 60.1; 51.5 | — |
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01833169). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.