Safety of BKM120 Monotherapy in Advanced Solid Tumor Patients

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01068483

Collaborator

(none)

107

Enrollment

Locations

Arm

Duration (Months)

21.4

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Breast Cancer Colon Cancer Ovarian Cancer Endometrium Cancer	Drug: BKM120	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

107 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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

Study Start Date :

Nov 1, 2008

Actual Primary Completion Date :

Sep 1, 2011

Actual Study Completion Date :

Aug 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: BKM120 Dose escalation followed by dose expansion	Drug: BKM120

Arm

Intervention/Treatment

Experimental: BKM120

Dose escalation followed by dose expansion

Drug: BKM120

Outcome Measures

Primary Outcome Measures

Maximum Tolerated Dose (MTD) of BKM120 [throughout the study]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Sarah Cannon Research Institute Dept.ofSarahCannonCancerCtr(4)	Nashville	Tennessee	United States	37203
2	Novartis Investigative Site	Toronto	Ontario	Canada	M5G 2M9
3	Novartis Investigative Site	Rotterdam		Netherlands	3015 CE
4	Novartis Investigative Site	Rotterdam		Netherlands	3075 EA
5	Novartis Investigative Site	Barcelona	Cataluna	Spain	08035