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Safety of BKM120 Monotherapy in Advanced Solid Tumor Patients

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: February 11, 2010
Last updated: April 9, 2013
Last verified: April 2013

This is a first-in-man, phase I clinical research study with BKM120, a potent and highly specific oral pan-class I phosphatidylinositol-3-kinase (PI3K) inhibitor. The study consists of a dose escalation part followed by a MTD expansion part.

Once the MTD has been defined, the MTD expansion part will be opened for enrollment.

Condition Intervention Phase
Breast Cancer
Colon Cancer
Ovarian Cancer
Endometrium Cancer
Drug: BKM120
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase IA, Multicenter, Open-label Dose Escalation Study of BKM120, Administered Orally in Adult Patients With Advanced Solid Malignancies

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Maximum Tolerated Dose (MTD) of BKM120 [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Safety and tolerability of BKM120 [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Pharmacokinetics of BKM120 [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Changes in tumor metabolic activity [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Pharmacodynamics of BKM120 [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Clinical tumor response in patients with tumors that show PI3K pathway activation [ Time Frame: throughout the study ] [ Designated as safety issue: No ]

Enrollment: 107
Study Start Date: November 2008
Study Completion Date: August 2012
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BKM120
Dose escalation followed by dose expansion
Drug: BKM120


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

All patients:

  • Histologically-confirmed advanced solid tumors
  • Progressive, recurrent unresectable disease
  • World Health Organization (WHO) Performance status ≤ 2
  • No history of primary brain tumor or brain metastases

MTD Expansion part:

  • Histologically-confirmed advanced solid tumors
  • Progressive, recurrent unresectable disease
  • World Health Organization (WHO) Performance status ≤ 2
  • No history of primary brain tumor; no brain metastases unless asymptomatic and clinically stable for ≥ 3 months
  • Patients with genetic or molecular alteration of the PI3K pathway

Exclusion Criteria:

  • Prior treatment with a PI3K inhibitor
  • History of or active major depressive episode, bipolar disorder, schizophrenia, or history of suicidal attempt or ideation
  • No clinically manifest diabetes mellitus (treated and/or with clinical signs)
  • No acute or chronic renal disease
  • No acute or chronic liver disease
  • No acute or chronic pancreatitis
  • No unresolved diarrhea
  • No impaired cardiac function or clinically significant cardiac diseases such as ventricular arrhythmia, congestive heart failure, uncontrolled hypertension
  • No acute myocardial infarction or unstable angina pectoris within the past 3 months
  • Not pregnant or nursing and fertile patients must use barrier contraceptives

Other protocol-defined inclusion/exclusion criteria may apply

  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 identifier: NCT01068483

United States, Tennessee
Sarah Cannon Research Institute Dept.ofSarahCannonCancerCtr(4)
Nashville, Tennessee, United States, 37203
Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M5G 2M9
Novartis Investigative Site
Rotterdam, Netherlands, 3015 CE
Novartis Investigative Site
Rotterdam, Netherlands, 3075 EA
Novartis Investigative Site
Barcelona, Cataluna, Spain, 08035
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT01068483     History of Changes
Other Study ID Numbers: CBKM120X2101  2008-002652-17 
Study First Received: February 11, 2010
Last Updated: April 9, 2013
Health Authority: United States: Food and Drug Administration
Spain: Ministry of Agriculture, Fisheries and Food
The Netherlands: Department of Public Healthand Environmental Protection
Canada: Health Canada

Keywords provided by Novartis:
Advanced solid tumors
Breast Cancer
Colon Cancer
Ovarian Cancer
Endometrium cancer

Additional relevant MeSH terms:
Ovarian Neoplasms
Colonic Neoplasms
Endometrial Neoplasms
Endocrine Gland Neoplasms
Neoplasms by Site
Ovarian Diseases
Adnexal Diseases
Genital Diseases, Female
Genital Neoplasms, Female
Urogenital Neoplasms
Endocrine System Diseases
Gonadal Disorders
Colorectal Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Digestive System Diseases
Gastrointestinal Diseases
Colonic Diseases
Intestinal Diseases
Uterine Neoplasms
Uterine Diseases processed this record on December 06, 2016