Clinical trial NCT01068483: Safety of BKM120 Monotherapy in Advanced Solid Tumor Patients
Official title: A Phase IA, Multicenter, Open-label Dose Escalation Study of BKM120, Administered Orally in Adult Patients With Advanced Solid Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Breast Cancer
Colon Cancer
Ovarian Cancer
Endometrium Cancer
Keywords: Advanced solid tumors
Breast Cancer
Colon Cancer
Ovarian Cancer
Endometrium cancer
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-02-15
Enrollment: 107
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Maximum Tolerated Dose (MTD) of BKM120
timeFrame: throughout the study
Secondary outcomes:
measure: Safety and tolerability of BKM120
timeFrame: throughout the study
measure: Pharmacokinetics of BKM120
timeFrame: throughout the study
measure: Changes in tumor metabolic activity
timeFrame: throughout the study
measure: Pharmacodynamics of BKM120
timeFrame: throughout the study
measure: Clinical tumor response in patients with tumors that show PI3K pathway activation
timeFrame: throughout the study
Other outcomes:
Locations:
city: Nashville
country: United States
facility: Sarah Cannon Research Institute Dept.ofSarahCannonCancerCtr(4)
state: Tennessee
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Rotterdam
country: Netherlands
facility: Novartis Investigative Site
city: Rotterdam
country: Netherlands
facility: Novartis Investigative Site
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Cataluna
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: