BKM120 for Patients With PI3K-activated Tumors

NCT01833169 | Completed Phase 2 Results

• 1 other identifier: CBKM120ZUS40
• Study Type: interventional
• Target: 146
• Locations: 1 country
• Sites: 59

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.

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

Outcome Measures

Primary Outcomes (1)

• Participant Clinical Benefit Response Rate

  Clinical benefit rate for patients with solid tumors will be assessed using RECIST 1.1 and will include responses of Complete Response (CR) or Partial Response (PR) or Stable Disease (SD) at \>=16 weeks. For hematologic tumors other appropriate hematological response criteria was applied. Response criteria: CR=Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm., PR=At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters, SD=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study, PD= At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline

  Week 16

Secondary Outcomes (7)

• 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

  baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months

• Progression-Free Survival - Number of Participants With an Event

  Every 8 Weeks until death, assessed up to 24 months

• Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months

  baseline up to 24 months

• Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages

  baseline up to 24 months

• Overall Survival - Number of Participants With an Event

  Every 8 Weeks until death, assessed up to 24 months

Study Arms (1)

BKM120

EXPERIMENTAL

BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28- day cycle

Drug: BKM120

Interventions

BKM120 DRUG

BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28-day cycle

BKM120

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (59)

Arizona Oncology Associates HOPE Division

Phoenix, Arizona, United States

Location

Arizona Oncology Associates PC- HAL

Sedona, Arizona, 86336, United States

Location

Highlands Oncology Group

Fayetteville, Arkansas, 72703, United States

Location

Sarcoma Oncology Center

Santa Monica, California, 90403, United States

Location

Rocky Mountain Cancer Centers RMCC - Aurora

Greenwood Village, Colorado, United States

Location

Whittingham Cancer Center Norwalk Hospital

Norwalk, Connecticut, 06856, United States

Location

Eastern Connecticut Hematology & Oncology Associates The Norwich Cancer Center

Norwich, Connecticut, 06360, United States

Location

Florida Cancer Specialists Dept of Oncology (2)

Fort Myers, Florida, 33901, United States

Location

Florida Hospital Cancer Institute FL Hosp. Cancer Instit.

Orlando, Florida, 32804, United States

Location

Florida Cancer Specialists

West Palm Beach, Florida, 33401, United States

Location

University Cancer & Blood Center, LLC

Athens, Georgia, 30607, United States

Location

Southeastern Regional Medical Center

Newnan, Georgia, 30265, United States

Location

Lurie Children's Hospital of Chicago Developmental Therapeutics

Chicago, Illinois, 60611, United States

Location

Cancer Care Specialists of Central Illinois

Decatur, Illinois, 62526, United States

Location

Illinois Cancer Care P.C. IL. Cancer Care

Peoria, Illinois, 61615-7828, United States

Location

Illinois Cancer Care P.C.

Peoria, Illinois, 61615-7828, United States

Location

Cancer Treatment Centers of America Southwestern Regional Med. Ctr

Schaumburg, Illinois, 60173, United States

Location

Northern Indiana Cancer Research Consortium No. Indiana Cancer Res.

South Bend, Indiana, 46617, United States

Location

University of Iowa Hospitals & Clinics Regulatory Contact 2

Iowa City, Iowa, 52242, United States

Location

Eastern Maine Medical Center Research Center SC

Bangor, Maine, 04401, United States

Location

Mount Auburn Hospital

Cambridge, Massachusetts, 02138, United States

Location

Michigan Medicine University of Michigan Int. Medicine Oncology

Ann Arbor, Michigan, 48109 5271, United States

Location

Cancer and Hematology Centers of West Michigan Dept. of Oncology

Grand Rapids, Michigan, 49546, United States

Location

Minnesota Oncology Hematology, P.A. Minnesota Oncology Hematology

Minneapolis, Minnesota, 55404, United States

Location

Nebraska Cancer Specialists Oncology Hematology West

Omaha, Nebraska, 68154, United States

Location

Comprehensive Cancer Centers

Las Vegas, Nevada, 89169, United States

Location

Hematology Oncology Associates of Northern New Jersey PA Regional Cancer Care Assoc.

Morristown, New Jersey, 07962, United States

Location

Cancer Center at Presbyterian

Albuquerque, New Mexico, United States

Location

New York Oncology Hematology NY Onc Hem

Albany, New York, 12208, United States

Location

Waverly Hematology Oncology

Cary, North Carolina, 27518, United States

Location

University of N C at Chapel Hill Physician Office Building

Chapel Hill, North Carolina, 27599-7600, United States

Location

Duke University Medical Center Seeley G. Mudd Bldg.

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic Foundation Cleveland Clinic (19)

Cleveland, Ohio, 44195, United States

Location

Mid Ohio Oncology/Hematology Mark H. Zangmeister Center

Columbus, Ohio, 43219, United States

Location

Bend Memorial Clinic Bend Mem. Clinic

Bend, Oregon, 97701, United States

Location

Northwest Cancer Specialists Compass Oncology (36)

Portland, Oregon, 97210, United States

Location

Oregon Health and Science University Oregon Health & Science U (56)

Portland, Oregon, 97239, United States

Location

Salem Health

Salem, Oregon, 97309, United States

Location

Gettysburg Cancer Center

Gettysburg, Pennsylvania, 17325, United States

Location

West Penn Allegheny Oncology Network

Natrona Heights, Pennsylvania, 15065, United States

Location

University of Pittsburgh Medical Center UPMC

Pittsburgh, Pennsylvania, 15213, United States

Location

Abington Hematology Oncology Associates, Inc Abington Hem Onc Assoc (5)

Willow Grove, Pennsylvania, 19090, United States

Location

Sanford University of South Dakota Medical Center Sanford Health

Sioux Falls, South Dakota, 57104, United States

Location

Chattanooga Oncology and Hematology Assoicates, PC Chattanooga Oncology

Chattanooga, Tennessee, 37404, United States

Location

West Cancer Clinic

Memphis, Tennessee, 38120, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Texas Oncology P A Round Rock

Dallas, Texas, 75251, United States

Location

Texas Oncology Sammons Cancer Center Sammons Cancer Center (10)

Dallas, Texas, 78246, United States

Location

Texas Oncology, P.A. Texas Oncololgy, P.A. (8)

Fort Worth, Texas, 76104, United States

Location

Oncology Consultants Oncology Group

Houston, Texas, 77024, United States

Location

MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)

Houston, Texas, 77030, United States

Location

Cancer Care Centers of South Texas HOAST CCC of So. TX-San Antonio (3)

San Antonio, Texas, 78229, United States

Location

Tyler Cancer Center

Tyler, Texas, 75702, United States

Location

Intermountain Medical Center Intermountain Healthcare

Murray, Utah, 84157, United States

Location

Virginia Cancer Specialists Virginia Cancer Specialists

Fairfax, Virginia, 22031, United States

Location

Blue Ridge Research Center at Roanoke Neurological Center McKesson Specialty Care

Roanoke, Virginia, 24018, United States

Location

Kadlec Clinic Hematology and Oncology Kadlec Clinic Hematology & Onc

Kennewick, Washington, 99336, United States

Location

Northwest Medical Specialties Hematology/Oncology

Tacoma, Washington, 98405, United States

Location

Medical College of Wisconsin Cancer Center

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Neoplasms; Hematologic Neoplasms; Leukemia, Myeloid, Acute

Interventions

NVP-BKM120

Condition Hierarchy (Ancestors)

Neoplasms by Site; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type

Limitations and Caveats

Secondary objective: Duration of response was not analyzed due to low response rate observed

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2013
• First Posted: April 16, 2013
• Study Start: March 29, 2013
• Primary Completion: September 26, 2016
• Study Completion: September 26, 2016
• Last Updated: October 19, 2018
• Results First Posted: December 12, 2017

Record last verified: 2018-10

Locations

US (59)