Safety of BKM120 Monotherapy in Advanced Solid Tumor Patients

Study Overview
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Brief Summary
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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.

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

Conditions
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Breast Cancer, Colon Cancer, Ovarian Cancer, Endometrium Cancer

Intervention / Treatment
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* Drug: BKM120


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BKM120<br>Dose escalation followed by dose expansion | Drug: BKM120<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum Tolerated Dose (MTD) of BKM120 |  | throughout the study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of BKM120 |  | throughout the study | 
| Pharmacokinetics of BKM120 |  | throughout the study | 
| Changes in tumor metabolic activity |  | throughout the study | 
| Pharmacodynamics of BKM120 |  | throughout the study | 
| Clinical tumor response in patients with tumors that show PI3K pathway activation |  | throughout the study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Advanced solid tumors, Breast Cancer, Colon Cancer, Ovarian Cancer, Endometrium cancer