BKM120 in Cancers With PIK3CA Activating Mutations

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01501604
Recruitment Status : Withdrawn (The study has been closed due to lack of accrual)
First Posted : December 29, 2011
Last Update Posted : September 17, 2015
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Jeffrey Engelman, Massachusetts General Hospital

December 27, 2011
December 29, 2011
September 17, 2015
January 2012
August 2013   (Final data collection date for primary outcome measure)
Response Rate [ Time Frame: 2 years ]
Objective Response Rate (CR or PR) by RECIST 1.1 criteria
Same as current
Complete list of historical versions of study NCT01501604 on Archive Site
  • Clinical Benefit Rate [ Time Frame: 2 years ]
    Clinical Benefit Rate (CR, PR, or SD) by RECIST 1.1 criteria
  • Survival [ Time Frame: 2 years ]
    Progression Free Survival (PFS)
  • Clinical Benefit [ Time Frame: 2 years ]
    Determine if the presence of specific co-existing mutations may influence clinical benefit from BKM120
Same as current
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BKM120 in Cancers With PIK3CA Activating Mutations
An Open Label, Phase II Trial of BKM120 in Cancers With PIK3CA Activating Mutations

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.

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
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Lung Cancer
  • Breast Cancer
  • Colorectal Cancer
  • Cholangiocarcinoma
  • Solid Tumors
Drug: BKM120
100 MG PO QD in cycles of 28 days
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
August 2013
August 2013   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
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United States
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Not Provided
Jeffrey Engelman, Massachusetts General Hospital
Massachusetts General Hospital
Novartis Pharmaceuticals
Principal Investigator: Jeffrey A Engelman, MD, PhD Massachusetts General Hospital
Massachusetts General Hospital
September 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP