BKM120 in Cancers With PIK3CA Activating Mutations

This study has been terminated.
(The study has been closed due to lack of accrual)
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Jeffrey Engelman, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT01501604
First received: December 27, 2011
Last updated: January 24, 2013
Last verified: January 2013
  Purpose

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 Intervention Phase
Lung Cancer
Breast Cancer
Colorectal Cancer
Cholangiocarcinoma
Solid Tumors
Drug: BKM120
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Phase II Trial of BKM120 in Cancers With PIK3CA Activating Mutations

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Response Rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Objective Response Rate (CR or PR) by RECIST 1.1 criteria


Secondary Outcome Measures:
  • Clinical Benefit Rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Clinical Benefit Rate (CR, PR, or SD) by RECIST 1.1 criteria

  • Survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Progression Free Survival (PFS)

  • Clinical Benefit [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Determine if the presence of specific co-existing mutations may influence clinical benefit from BKM120


Estimated Enrollment: 40
Study Start Date: January 2012
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: BKM120
    100 MG PO QD in cycles of 28 days
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
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01501604

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

No publications provided

Responsible Party: Jeffrey Engelman, Director, Thoracic Oncology, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT01501604     History of Changes
Other Study ID Numbers: 11-211
Study First Received: December 27, 2011
Last Updated: January 24, 2013
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Breast Neoplasms
Colorectal Neoplasms
Lung Neoplasms
Cholangiocarcinoma
Neoplasms by Site
Neoplasms
Breast Diseases
Skin Diseases
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Digestive System Diseases
Gastrointestinal Diseases
Colonic Diseases
Intestinal Diseases
Rectal Diseases
Respiratory Tract Neoplasms
Thoracic Neoplasms
Lung Diseases
Respiratory Tract Diseases
Adenocarcinoma
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type

ClinicalTrials.gov processed this record on August 25, 2014