Ignite Creation Date:
2025-12-24 @ 9:26 PM
Ignite Modification Date:
2026-01-02 @ 9:40 AM
Study NCT ID:
NCT01790932
Status:
COMPLETED
Last Update Posted:
2019-01-23
First Post:
2013-02-07
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
BKM120 For Triple Negative Breast Cancer
Sponsor:
Dana-Farber Cancer Institute
Organization:
Study Overview
Official Title:
A Phase II Trial of BKM120 in Patients With Triple Negative Metastatic Breast Cancer
Status:
COMPLETED
Status Verified Date:
2019-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Triple negative breast cancer (TNBC) has an aggressive phenotype and poor prognosis. This tumor type characterized by lack of expression of estrogen receptor (ER), progesterone receptor (PR) and no amplification of the human epidermal growth factor 2 (HER2) accounts for 15% of breast cancers. Limited treatment options exist in the clinic as hormonal therapies and HER2-trageted agents have proven ineffective. BKM120 is a drug that works by blocking a protein called phosphatidylinositol-3-kinase (PI3K) which may contribute to cancer growth. This drug has been used in experiments in the laboratory and information from these research studies suggests that BKM120 may help to prevent cancer cells from growing. In this research study, the investigators are looking to see if BKM120 works to stop breast cancer cells from growing.
Detailed Description:
Study plan Two investigator-initiated protocols (one for US, one for Spain) will be enrolling in parallel. The first 50 participants will be recruited concurrently in US and Spain.
Stage 1 Stage 1 will include up to 50 participants with advanced TN disease. Available tumor block is required in all participants per inclusion criteria. Analysis of this tumor block will be used for correlation of predictive markers and clinical response in order to define potential subpopulation that benefit from BKM120. In Stage 1, all participants will have biopsies done at baseline, cycle 1 day 28/cycle 2 day 1 and end of treatment to analyze drug effect in the PI3K and mitogen-activated protein kinases (MAPK) pathway. This will aid to understand the pharmacodynamic effects of BKM120 in tumors with similar genetic background (triple negative disease). The enrollment of Stage 1 will ensure that at least 10 paired evaluable biopsies are obtained. After the enrollment of the first 29 evaluable subjects enrolled overall in Stage 1 (considering the US and the Spanish protocol), the Steering Committee will perform an interim analysis of safety and efficacy. If absolutely no activity is observed, the clinical trial will close and no more subjects will be enrolled. If there are early signs of activity (one patient or more achieving clinical benefit response), enrollment will proceed until 50 participants are enrolled in Stage 1. After 50 patients have been enrolled, we will analyze preliminary responses to treatment depending on the molecular status of each patient.
Stage 2 Were the trial to continue at the end of Stage 1, 50 participants would have been treated and their clinical status and response to therapy will be available. Also, paraffin blocks from these participants will have been analyzed for predictive markers of treatment effect. If there is clinical activity observed in Stage 1 and this analysis shows preliminary signs of response in a subpopulation based on the presence or absence of tumor PI3K pathway alterations, participant pre-selection may be implemented for Stage 2 (justified in an amendment before proceeding to Stage 2.
Stage 1 Stage 1 will include up to 50 participants with advanced TN disease. Available tumor block is required in all participants per inclusion criteria. Analysis of this tumor block will be used for correlation of predictive markers and clinical response in order to define potential subpopulation that benefit from BKM120. In Stage 1, all participants will have biopsies done at baseline, cycle 1 day 28/cycle 2 day 1 and end of treatment to analyze drug effect in the PI3K and mitogen-activated protein kinases (MAPK) pathway. This will aid to understand the pharmacodynamic effects of BKM120 in tumors with similar genetic background (triple negative disease). The enrollment of Stage 1 will ensure that at least 10 paired evaluable biopsies are obtained. After the enrollment of the first 29 evaluable subjects enrolled overall in Stage 1 (considering the US and the Spanish protocol), the Steering Committee will perform an interim analysis of safety and efficacy. If absolutely no activity is observed, the clinical trial will close and no more subjects will be enrolled. If there are early signs of activity (one patient or more achieving clinical benefit response), enrollment will proceed until 50 participants are enrolled in Stage 1. After 50 patients have been enrolled, we will analyze preliminary responses to treatment depending on the molecular status of each patient.
Stage 2 Were the trial to continue at the end of Stage 1, 50 participants would have been treated and their clinical status and response to therapy will be available. Also, paraffin blocks from these participants will have been analyzed for predictive markers of treatment effect. If there is clinical activity observed in Stage 1 and this analysis shows preliminary signs of response in a subpopulation based on the presence or absence of tumor PI3K pathway alterations, participant pre-selection may be implemented for Stage 2 (justified in an amendment before proceeding to Stage 2.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: