Safety of BKM120 Monotherapy in Advanced Solid Tumor Patients

A Phase IA, Multicenter, Open-label Dose Escalation Study of BKM120, Administered Orally in Adult Patients With Advanced Solid Malignancies

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.

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