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BKM120 in Cancers With PIK3CA Activating Mutations

Primary Purpose

Lung Cancer, Breast Cancer, Colorectal Cancer

Status

Withdrawn

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

BKM120

About this trial

This is an interventional treatment trial for Lung Cancer focused on measuring PIK3CA mutation, Advanced solid tumors

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

At least 1 site of measurable disease
Life expectancy >/= 12 weeks
Adequate marrow and organ function
Diagnosis of lung cancer, breast cancer, colorectal cancer, cholangiocarcinoma, gastric cancer, pancreatic cancer, prostate cancer, uterine cancer, ovarian cancer, esophageal cancer, or head and neck cancer
Pathologically documented, definitively diagnosed, advanced solid tuor that is refractory to standard treatment, for which no standard therapy is available, or the subject refuses standard therapy
Cancer must have at least one of the following PIK3CA mutations: E542K, E545K, H1047R, H1047L. The PIK3CA mutation must be documented in a CLIA approved laboratory

Exclusion Criteria:

Prior treatment with a P13K inhibitor
Known hypersensitivity to BKM120 or its excipients
Untreated brain metastases
Acute or chronic liver, renal disease or pancreatitis
Currently treated with drugs known to be moderate and strong inhibitors or inducers of isoenzyme CYP3A
Diarrhea >/= CTCAE grade 2
Any concurrent severe and/or uncontrolled medical condition
Active cardiac disease
History of cardiac dysfunction
Poorly controlled diabetes mellitus or steroid-induced diabetes mellitus
Significant symptomatic deterioration of lung function
Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of BKDM120 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
Currently taking therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulant
Pregnant or breast-feeding
Known diagnosis of HIV infection
History of another malignancy within 3 years, except cured basal cell carcinoma of the skin or excised carcinoma in situ of the cervix
Unable to swallow the medication in its prescribed form

Sites / Locations

Outcomes

Primary Outcome Measures

Response Rate

Objective Response Rate (CR or PR) by RECIST 1.1 criteria

Secondary Outcome Measures

Clinical Benefit Rate

Clinical Benefit Rate (CR, PR, or SD) by RECIST 1.1 criteria

Survival

Progression Free Survival (PFS)

Clinical Benefit

Determine if the presence of specific co-existing mutations may influence clinical benefit from BKM120

Full Information

NCT ID

NCT01501604

First Posted

December 27, 2011

Last Updated

September 16, 2015

Sponsor

Massachusetts General Hospital

Collaborators

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT01501604

Brief Title

BKM120 in Cancers With PIK3CA Activating Mutations

Official Title

An Open Label, Phase II Trial of BKM120 in Cancers With PIK3CA Activating Mutations

Study Type

Interventional

2. Study Status

Record Verification Date

September 2015

Overall Recruitment Status

Withdrawn

Why Stopped

The study has been closed due to lack of accrual

Study Start Date

January 2012 (undefined)

Primary Completion Date

August 2013 (Actual)

Study Completion Date

August 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Massachusetts General Hospital

Collaborators

Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

In people whos cancers have a PIK3CA mutation, this trial will be evaluating the drug BKM120 as a possible treatment. BKM120 works by blocking the phosphatidylinositol-3-kinase (PI3K)pathway, thereby inhibiting tumor growth and survival. The purpose of this study is to learn if the study drug BKM120 can shrink or slow the growth of your tumor. The safety of BKM120 will also be studied. Your physical state, symptoms, change in the size of your tumor, and laboratory findings obtained while you are on study will help the research team decide if BKM120 is safe and effective in patients with advanced cancers.

Detailed Description

Subjects enrolled in this study will receive BKM120 once daily, orally, in cycles of 28 days. During Cycles 1 and 2, the following tests and procedures will be done on days 1 and 15: physical exam performance status blood tests pregnancy test (if applicable) neuropsychiatric assessments Starting at Cycle 2 and then every other cycle thereafter (approximately every 8 weeks) tumor assessment will be performed by CT/MRI or PET scan. A chest x-ray will also be performed every 8 weeks. Beginning with Cycle 3, the following tests/procedures will be performed on Day 1 of each cycle: physical exam performance status blood tests neuropsychiatric assessments

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Lung Cancer, Breast Cancer, Colorectal Cancer, Cholangiocarcinoma, Solid Tumors

Keywords

PIK3CA mutation, Advanced solid tumors

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

BKM120

Intervention Description

100 MG PO QD in cycles of 28 days

Primary Outcome Measure Information:

Title

Response Rate

Description

Objective Response Rate (CR or PR) by RECIST 1.1 criteria

Time Frame

2 years

Secondary Outcome Measure Information:

Title

Clinical Benefit Rate

Description

Clinical Benefit Rate (CR, PR, or SD) by RECIST 1.1 criteria

Time Frame

2 years

Title

Survival

Description

Progression Free Survival (PFS)

Time Frame

2 years

Title

Clinical Benefit

Description

Determine if the presence of specific co-existing mutations may influence clinical benefit from BKM120

Time Frame

2 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: At least 1 site of measurable disease Life expectancy >/= 12 weeks Adequate marrow and organ function Diagnosis of lung cancer, breast cancer, colorectal cancer, cholangiocarcinoma, gastric cancer, pancreatic cancer, prostate cancer, uterine cancer, ovarian cancer, esophageal cancer, or head and neck cancer Pathologically documented, definitively diagnosed, advanced solid tuor that is refractory to standard treatment, for which no standard therapy is available, or the subject refuses standard therapy Cancer must have at least one of the following PIK3CA mutations: E542K, E545K, H1047R, H1047L. The PIK3CA mutation must be documented in a CLIA approved laboratory Exclusion Criteria: Prior treatment with a P13K inhibitor Known hypersensitivity to BKM120 or its excipients Untreated brain metastases Acute or chronic liver, renal disease or pancreatitis Currently treated with drugs known to be moderate and strong inhibitors or inducers of isoenzyme CYP3A Diarrhea >/= CTCAE grade 2 Any concurrent severe and/or uncontrolled medical condition Active cardiac disease History of cardiac dysfunction Poorly controlled diabetes mellitus or steroid-induced diabetes mellitus Significant symptomatic deterioration of lung function Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of BKDM120 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection) Currently taking therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulant Pregnant or breast-feeding Known diagnosis of HIV infection History of another malignancy within 3 years, except cured basal cell carcinoma of the skin or excised carcinoma in situ of the cervix Unable to swallow the medication in its prescribed form

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jeffrey A Engelman, MD, PhD

Organizational Affiliation

Massachusetts General Hospital

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

BKM120 in Cancers With PIK3CA Activating Mutations

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