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

Phase 2
18 Years
Not Enrolling
Lung Cancer, Breast Cancer, Colorectal Cancer, Cholangiocarcinoma, Solid Tumors

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Trial Information

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

Subjects enrolled in this study will receive BKM120 once daily, orally, in cycles of 28

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

- physical exam

- performance status

- blood tests

- neuropsychiatric assessments

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response Rate

Outcome Description:

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

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Jeffrey A Engelman, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

January 2012

Completion Date:

December 2015

Related Keywords:

  • Lung Cancer
  • Breast Cancer
  • Colorectal Cancer
  • Cholangiocarcinoma
  • Solid Tumors
  • PIK3CA mutation
  • Advanced solid tumors
  • Breast Neoplasms
  • Colorectal Neoplasms
  • Lung Neoplasms
  • Cholangiocarcinoma