Clinical trial NCT01297491: Safety and Efficacy of BKM120 in Patients With Metastatic Non-small Cell Lung Cancer
Official title: An Open Label Two-stage Study of Orally Administered BKM120 in Patients With Metastatic Non-small Cell Lung Cancer With Activated PI3K Pathway
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Non-small Cell Lung Cancer
Keywords: NSCLC
PI3K
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-02-16
Enrollment: 63
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this two-stage phase II study is to assess the efficacy of BKM120, as measured by determining the progression free survival (PFS), in patients with pretreated metastatic Non-small Cell Lung Cancer (NSCLC) that exhibits PI3K pathway activation. BKM120 will be investigated in two groups of NSCLC patients according to the histology of the cancer: squamous and non-squamous.


Eligibility criteria: Inclusion Criteria:

* Histologically confirmed NSCLC with activated PI3K pathway
* Progressive disease after prior systemic antineoplastic treatment(s) for advanced NSCLC
* Archival or fresh tumor biopsy must be available for profiling
* Measurable and/or non-measurable disease as per RECIST 1.1 criteria
* Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
* Adequate organ function as assessed by laboratory tests

Exclusion Criteria:

* Patient has received previous treatment with PI3K inhibitors
* Patient with squamous NSCLC has received more than one line of chemotherapy treatment for metastatic disease; patient with non-squamous NSCLC has received more than two lines of systemic antineoplastic treatment for metastatic disease
* Uncontrolled or symptomatic CNS metastases
* Concurrent use of any other approved or investigational antineoplastic agent
* Radiotherapy ≤ 28 days prior to starting study drug
* Major surgery within 28 days prior to starting study drug
* History of clinically significant cardiac dysfunction, mood disorders, or poorly controlled diabetes mellitus
* Current treatment with medication that has a known risk to prolong the QT interval or inducing Torsades de Pointes
* Impairment of gastrointestinal (GI) function
* Chronic treatment with steroids or another immunosuppressive agent.
* Concurrent severe and/or uncontrolled medical condition
* Currently receiving Warfarin or another coumarin derivative
* Known history of HIV infection
* Sensory neuropathy with functional impairment (CTC grade 2 neuropathy, regardless of causality)
* Pregnancy, lactation, or breastfeeding
* Woman of child-bearing potential

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Progression Free Survival (PFS) Rate as Per Investigator Local Review Measured Using RECIST 1.1 of Patients at Week 12
timeFrame: Week 12
Secondary outcomes:
measure: Overall Survival (OS) Using Kaplan-Meier Estimates
timeFrame: Every 8 weeks up to 24 months
measure: Overall Response Rate (ORR) Based on Investigator Assessment
timeFrame: Every 6 weeks up to 24 months
measure: Disease Control Rate (DCR)
timeFrame: Every 6 weeks up tp 24 months
measure: Time to Response (TTR)
timeFrame: Every 6 weeks up to 24 months
measure: Duration of Response (DoR)
timeFrame: Every 6 weeks up to 24 months
Other outcomes:
Locations:
city: Chandler
country: United States
facility: Ironwood Cancer and Research Centers SC
state: Arizona
city: Phoenix
country: United States
facility: Arizona Oncology Associates Tucson (Rudasill & La Cholla)
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic - Arizona Mayo Scottsdale AZ
state: Arizona
city: Fayetteville
country: United States
facility: Highlands Oncology Group Dept of Highlands Oncology Grp
state: Arkansas
city: Los Angeles
country: United States
facility: Cedars Sinai Medical Center Dept.of Cedars-Sinai Med. Ctr.
state: California
city: San Diego
country: United States
facility: University of California at San Diego, Moores Cancer Ctr SC
state: California
city: Aurora
country: United States
facility: University of Colorado Univ CO
state: Colorado
city: Greenwood Village
country: United States
facility: Rocky Mountain Cancer Centers Dept. of Rocky Mountain Cancer
state: Colorado
city: Tampa
country: United States
facility: H. Lee Moffitt Cancer Center & Research Institute H Lee Moffitt
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine/Winship Cancer Institute Emory 2
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center SC
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center Unvi Chi
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Cancer Center Univ of KS
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital Mass General
state: Massachusetts
city: Worcester
country: United States
facility: Fallon Clinic at Worcester Medical Center Fallon Clinic Worcester Med
state: Massachusetts
city: Detroit
country: United States
facility: Karmanos Cancer Institute Wayne St Karmanos
state: Michigan
city: St. Louis
country: United States
facility: Washington University School of Medicine Washington University (16)
state: Missouri
city: Morristown
country: United States
facility: Hematology Oncology Associates of Northern New Jersey PA DeptofHem-OncofNorthern NJ (2)
state: New Jersey
city: Summit
country: United States
facility: Overlook Hospital - Carol G Simon Cancer Center Carol G Simon
state: New Jersey
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute Rosewell
state: New York
city: NY
country: United States
facility: Memorial Sloan Kettering Cancer Center Sloan Kettering
state: New York
city: Durham
country: United States
facility: Duke University Medical Center Duke 2
state: North Carolina
city: Cleveland
country: United States
facility: MetroHealth Medical Center Dept.ofMetroHealthMedCtr.(2)
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center Dept. of Oklahoma Univ. HSC
state: Oklahoma
city: Portland
country: United States
facility: Northwest Cancer Specialists Compass Oncology -BKM
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center SC-2
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina MUSC
state: South Carolina
city: Dallas
country: United States
facility: Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology
state: Texas
city: Dallas
country: United States
facility: Texas Oncology South Texas Oncology
state: Texas
city: Dallas
country: United States
facility: U of TX Southwestern Medical Center - SimmonsCompCancerCtr Clinical Research Office
state: Texas
city: *see Various Departments*
country: United States
facility: Virginia Oncology Associates VOA - Lake Wright (2)
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin Univ WIsc 2
state: Wisconsin
city: Rio Negro
country: Argentina
facility: Novartis Investigative Site
state: Viedma
city: Buenos Aires
country: Argentina
facility: Novartis Investigative Site
city: Cordoba
country: Argentina
facility: Novartis Investigative Site
city: Brussel
country: Belgium
facility: Novartis Investigative Site
city: Charleroi
country: Belgium
facility: Novartis Investigative Site
city: Genk
country: Belgium
facility: Novartis Investigative Site
city: Leuven
country: Belgium
facility: Novartis Investigative Site
city: Libramont
country: Belgium
facility: Novartis Investigative Site
city: Salvador
country: Brazil
facility: Novartis Investigative Site
state: BA
city: Rio de Janeiro
country: Brazil
facility: Novartis Investigative Site
state: RJ
city: Florianopolis
country: Brazil
facility: Novartis Investigative Site
state: SC
city: Barretos
country: Brazil
facility: Novartis Investigative Site
state: SP
city: São Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Vancouver
country: Canada
facility: Novartis Investigative Site
state: British Columbia
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Caen Cedex
country: France
facility: Novartis Investigative Site
city: Creteil
country: France
facility: Novartis Investigative Site
city: Marseille cedex 20
country: France
facility: Novartis Investigative Site
city: Rennes
country: France
facility: Novartis Investigative Site
city: Villejuif Cedex
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Gauting
country: Germany
facility: Novartis Investigative Site
city: Grosshansdorf
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
city: Nuernberg
country: Germany
facility: Novartis Investigative Site
city: Oldenburg
country: Germany
facility: Novartis Investigative Site
city: Recklinghausen
country: Germany
facility: Novartis Investigative Site
city: Hongkong
country: Hong Kong
facility: Novartis Investigative Site
city: Budapest
country: Hungary
facility: Novartis Investigative Site
city: Budapest
country: Hungary
facility: Novartis Investigative Site
city: Deszk
country: Hungary
facility: Novartis Investigative Site
city: Mátraháza
country: Hungary
facility: Novartis Investigative Site
city: Szolnok
country: Hungary
facility: Novartis Investigative Site
city: Avellino
country: Italy
facility: Novartis Investigative Site
state: AV
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Parma
country: Italy
facility: Novartis Investigative Site
state: PR
city: Udine
country: Italy
facility: Novartis Investigative Site
state: UD
city: Kurashiki
country: Japan
facility: Novartis Investigative Site
state: Okayama
city: Koto
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Maastricht
country: Netherlands
facility: Novartis Investigative Site
city: Singapore
country: Singapore
facility: Novartis Investigative Site
city: Sabadell
country: Spain
facility: Novartis Investigative Site
state: Barcelona
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Mataro
country: Spain
facility: Novartis Investigative Site
state: Cataluña
city: Alicante
country: Spain
facility: Novartis Investigative Site
state: Comunidad Valenciana
city: Tainan 704
country: Taiwan
facility: Novartis Investigative Site
state: Taiwan ROC
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
state: Taiwan ROC
city: Bangkok
country: Thailand
facility: Novartis Investigative Site
city: Chiang Mai
country: Thailand
facility: Novartis Investigative Site
city: Altunizade
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: Northwood
country: United Kingdom
facility: Novartis Investigative Site
state: Middlesex
city: Leicester
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: Manchester
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Vansteenkiste JF, Canon JL, De Braud F, Grossi F, De Pas T, Gray JE, Su WC, Felip E, Yoshioka H, Gridelli C, Dy GK, Thongprasert S, Reck M, Aimone P, Vidam GA, Roussou P, Wang YA, Di Tomaso E, Soria JC. Safety and Efficacy of Buparlisib (BKM120) in Patients with PI3K Pathway-Activated Non-Small Cell Lung Cancer: Results from the Phase II BASALT-1 Study. J Thorac Oncol. 2015 Sep;10(9):1319-1327. doi: 10.1097/JTO.0000000000000607.