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Recruitment Status : Withdrawn (The study has been closed due to lack of accrual)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

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.

Condition or disease Intervention/treatment Phase
Lung Cancer Breast Cancer Colorectal Cancer Cholangiocarcinoma Solid Tumors Drug: BKM120 Phase 2

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Phase II Trial of BKM120 in Cancers With PIK3CA Activating Mutations
Study Start Date : January 2012
Actual Primary Completion Date : August 2013
Actual Study Completion Date : August 2013

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: BKM120
    100 MG PO QD in cycles of 28 days

Primary Outcome Measures :
  1. Response Rate [ Time Frame: 2 years ]
    Objective Response Rate (CR or PR) by RECIST 1.1 criteria

Secondary Outcome Measures :
  1. Clinical Benefit Rate [ Time Frame: 2 years ]
    Clinical Benefit Rate (CR, PR, or SD) by RECIST 1.1 criteria

  2. Survival [ Time Frame: 2 years ]
    Progression Free Survival (PFS)

  3. Clinical Benefit [ Time Frame: 2 years ]
    Determine if the presence of specific co-existing mutations may influence clinical benefit from BKM120

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01501604

Sponsors and Collaborators
Massachusetts General Hospital
Novartis Pharmaceuticals
Principal Investigator: Jeffrey A Engelman, MD, PhD Massachusetts General Hospital

Responsible Party: Jeffrey Engelman, Director, Thoracic Oncology, Massachusetts General Hospital Identifier: NCT01501604     History of Changes
Other Study ID Numbers: 11-211
First Posted: December 29, 2011    Key Record Dates
Last Update Posted: September 17, 2015
Last Verified: September 2015

Keywords provided by Jeffrey Engelman, Massachusetts General Hospital:
PIK3CA mutation
Advanced solid tumors

Additional relevant MeSH terms:
Colorectal Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Colonic Diseases
Intestinal Diseases
Rectal Diseases
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type