Clinical trial NCT01833169: BKM120 for Patients With PI3K-activated Tumors
Official title: Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 1 - BKM120 for Patients With PI3K-activated Tumors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: PI3K Pathway Activated Tumors
Keywords: Solid tumor
malignancy
hematologic malignancy
PI3K pathway activation
P13K activated tumors
BKM120
signature
AML
acute myelogenous leukemia
cervical
ovarian
hepatobiliary
buparlisib
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-04-16
Enrollment: 146
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this signal seeking study was is to determine whether treatment with BKM120 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.


Eligibility criteria: Inclusion Criteria:

* Patient had a confirmed diagnosis of a solid tumor or hematological malignancy with the exception of endometrial cancer, glioblastoma, nonsmall cell lung cancer, prostate cancer or breast cancer.
* Patient's tumor was evaluated and pre-identified to have activation of the PI3K pathway, at a CLIA certified laboratory
* Patient must have received at least one prior treatment for recurrent metastatic and /or locally advanced disease and for whom no standard therapy options was anticipated to result in a durable remission.
* Patient must have had progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines
* Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

Exclusion Criteria:

Patient had received previous treatment with BKM120 Patient had symptomatic CNS metastases Patient had mood disorder as outlined in Section 5 Patient had received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.

Primary outcomes:
measure: Participant Clinical Benefit Response Rate
timeFrame: Week 16
Secondary outcomes:
measure: Overall Response of Partial Response (PR) or Greater. PR=at Least a 30% Decrease in the Sum of Diameters of Target Lesions, Taking as Reference the Baseline Sum Diameters
timeFrame: baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months
measure: Progression-Free Survival - Number of Participants With an Event
timeFrame: Every 8 Weeks until death, assessed up to 24 months
measure: Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months
timeFrame: baseline up to 24 months
measure: Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages
timeFrame: baseline up to 24 months
measure: Overall Survival - Number of Participants With an Event
timeFrame: Every 8 Weeks until death, assessed up to 24 months
measure: Overall Survival (OS)- Kaplan-Meier Estimates of OS Timing in Months
timeFrame: baseline up to 24 months
measure: Overall Survival (OS)- Kaplan-Meier Estimates of OS Rate in Percentages
timeFrame: baseline up to 30 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona Oncology Associates HOPE Division
state: Arizona
city: Sedona
country: United States
facility: Arizona Oncology Associates PC- HAL
state: Arizona
city: Fayetteville
country: United States
facility: Highlands Oncology Group
state: Arkansas
city: Santa Monica
country: United States
facility: Sarcoma Oncology Center
state: California
city: Greenwood Village
country: United States
facility: Rocky Mountain Cancer Centers RMCC - Aurora
state: Colorado
city: Norwalk
country: United States
facility: Whittingham Cancer Center Norwalk Hospital
state: Connecticut
city: Norwich
country: United States
facility: Eastern Connecticut Hematology & Oncology Associates The Norwich Cancer Center
state: Connecticut
city: Fort Myers
country: United States
facility: Florida Cancer Specialists Dept of Oncology (2)
state: Florida
city: Orlando
country: United States
facility: Florida Hospital Cancer Institute FL Hosp. Cancer Instit.
state: Florida
city: West Palm Beach
country: United States
facility: Florida Cancer Specialists
state: Florida
city: Athens
country: United States
facility: University Cancer & Blood Center, LLC
state: Georgia
city: Newnan
country: United States
facility: Southeastern Regional Medical Center
state: Georgia
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago Developmental Therapeutics
state: Illinois
city: Decatur
country: United States
facility: Cancer Care Specialists of Central Illinois
state: Illinois
city: Peoria
country: United States
facility: Illinois Cancer Care P.C. IL. Cancer Care
state: Illinois
city: Peoria
country: United States
facility: Illinois Cancer Care P.C.
state: Illinois
city: Schaumburg
country: United States
facility: Cancer Treatment Centers of America Southwestern Regional Med. Ctr
state: Illinois
city: South Bend
country: United States
facility: Northern Indiana Cancer Research Consortium No. Indiana Cancer Res.
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics Regulatory Contact 2
state: Iowa
city: Bangor
country: United States
facility: Eastern Maine Medical Center Research Center SC
state: Maine
city: Cambridge
country: United States
facility: Mount Auburn Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: Michigan Medicine University of Michigan Int. Medicine Oncology
state: Michigan
city: Grand Rapids
country: United States
facility: Cancer and Hematology Centers of West Michigan Dept. of Oncology
state: Michigan
city: Minneapolis
country: United States
facility: Minnesota Oncology Hematology, P.A. Minnesota Oncology Hematology
state: Minnesota
city: Omaha
country: United States
facility: Nebraska Cancer Specialists Oncology Hematology West
state: Nebraska
city: Las Vegas
country: United States
facility: Comprehensive Cancer Centers
state: Nevada
city: Morristown
country: United States
facility: Hematology Oncology Associates of Northern New Jersey PA Regional Cancer Care Assoc.
state: New Jersey
city: Albuquerque
country: United States
facility: Cancer Center at Presbyterian
state: New Mexico
city: Albany
country: United States
facility: New York Oncology Hematology NY Onc Hem
state: New York
city: Cary
country: United States
facility: Waverly Hematology Oncology
state: North Carolina
city: Chapel Hill
country: United States
facility: University of N C at Chapel Hill Physician Office Building
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center Seeley G. Mudd Bldg.
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Cleveland Clinic (19)
state: Ohio
city: Columbus
country: United States
facility: Mid Ohio Oncology/Hematology Mark H. Zangmeister Center
state: Ohio
city: Bend
country: United States
facility: Bend Memorial Clinic Bend Mem. Clinic
state: Oregon
city: Portland
country: United States
facility: Northwest Cancer Specialists Compass Oncology (36)
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University Oregon Health & Science U (56)
state: Oregon
city: Salem
country: United States
facility: Salem Health
state: Oregon
city: Gettysburg
country: United States
facility: Gettysburg Cancer Center
state: Pennsylvania
city: Natrona Heights
country: United States
facility: West Penn Allegheny Oncology Network
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center UPMC
state: Pennsylvania
city: Willow Grove
country: United States
facility: Abington Hematology Oncology Associates, Inc Abington Hem Onc Assoc (5)
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sanford University of South Dakota Medical Center Sanford Health
state: South Dakota
city: Chattanooga
country: United States
facility: Chattanooga Oncology and Hematology Assoicates, PC Chattanooga Oncology
state: Tennessee
city: Memphis
country: United States
facility: West Cancer Clinic
state: Tennessee
city: Nashville
country: United States
facility: Sarah Cannon Research Institute
state: Tennessee
city: Dallas
country: United States
facility: Texas Oncology P A Round Rock
state: Texas
city: Dallas
country: United States
facility: Texas Oncology Sammons Cancer Center Sammons Cancer Center (10)
state: Texas
city: Fort Worth
country: United States
facility: Texas Oncology, P.A. Texas Oncololgy, P.A. (8)
state: Texas
city: Houston
country: United States
facility: Oncology Consultants Oncology Group
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)
state: Texas
city: San Antonio
country: United States
facility: Cancer Care Centers of South Texas HOAST CCC of So. TX-San Antonio (3)
state: Texas
city: Tyler
country: United States
facility: Tyler Cancer Center
state: Texas
city: Murray
country: United States
facility: Intermountain Medical Center Intermountain Healthcare
state: Utah
city: Fairfax
country: United States
facility: Virginia Cancer Specialists Virginia Cancer Specialists
state: Virginia
city: Roanoke
country: United States
facility: Blue Ridge Research Center at Roanoke Neurological Center McKesson Specialty Care
state: Virginia
city: Kennewick
country: United States
facility: Kadlec Clinic Hematology and Oncology Kadlec Clinic Hematology & Onc
state: Washington
city: Tacoma
country: United States
facility: Northwest Medical Specialties Hematology/Oncology
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin Cancer Center
state: Wisconsin
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: