UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase 2 study of BKM120 in patients with advanced esophagus cancer
Title of the study (Brief title)	BKM120 Phase 2 ESCC
Region	Japan

Condition
Condition	advanced esophagial cancer
Classification by specialty	Gastroenterology
Classification by malignancy	Malignancy
Genomic information	YES

Objectives
Narrative objectives1	To evaluate the efficacy and safety of BKM120 in patients with advanced esophagus cancer which refractory to pre-chemotherpy.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	Disease control rate (DCR)
Key secondary outcomes	Response rate: RR Progression free survival: PFS Overall survival : OS Adverse event : AE

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	BKM120 is administered orally once daily in 28-days cycles.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Histologically proven esophagus squamous cancer. 2) With inoperable, recurrence, or metastatic lesion. 3)Prior chemotherapy was discontinued according to disease progression, or an adverse event. 4)>= 20 years old. 5)ECOG Performance Status of 0-1, or 2. 6)With one or more measurable lesion (based on a RECIST guideline ver1.1.) 7)Prior chemotherapies meet the criteria as follows. 1.have a history of a fluorinated Pyrimidine agents. 2.have a history of a platinum Agents. 3.History of 2 or more regimens. 8)Adequate organ function. I.e. meet all criterions as follows. 1.Absolute neutrophil count >=1,500 /mm3 2.Hemoglobin >=9.0 g/dL 3.Platelet count >=100,000 /mm3 4.Total bilirubin <=1.2 mg/dL 5.AST<=40 IU/L or <=120 IU/L with liver metastasis. 6.ALT <=40 IU/L or <=120 IU/L with liver metastasis. 7.Serum creatinine male <=1.5mg/dL female<=1.0mg/dL 8.Blood sugar <=120 mg/dL 9.HbA1c<=6.5% 9)No blood transfusion within 14 days prior to enrollment. 10)AE related to prior chemotherapy, radiation therapy or surgery must be Grade <= 1 according to CTCAE. 11)Women of childbearing potential must have a negative serum pregnancy test within 14 days prior to enrollment. 12)Adequate oral intake. 13)Written informed consent. 14)Tumor tissue is available for a cancer-related somatic gene analysis.
Key exclusion criteria	1)Prior chemotherapy within 3 weeks prior to enrollment. 2)Major Surgery or radiation therapy within 3 weeks prior to enrollment. 3)Other investigational drugs within 3 weeks prior to enrollment. 4)with the history of PI3K Inhibitor AKT Inhibitor or mTOR Inhibitor treatment. 5)With CNS metastasis. 6)Carcinomatous pleural effusion, or ascites, cardiac effusion with indication of invasive treatment. 7)Synchronous or asynchronous other cancer except carcinoma in situ or intramucosal carcinoma. 8)Any severe and / or uncontrolled medical conditions. 9)Interstitial pneumonia. 10)Positive HBs antigen,HCV antibodyor known positive HIV antibody test. 11)Chronic treatment with steroids or another immunosuppressive agent. 12)Chronic treatment with warfarin or coumarin derivatives. 13)Treatment with strong CYP3A inhibitor. 14)PHQ-9 questionnaire is 12 or more. 15)PHQ-9 No.9 concerning the possibility of suicide idea of the PHQ-9 questionnaire was answered, "1,2 or 3". 16)GAD-7 questionnaire is >=15 17)The major depressive episode, the bipolar disorder,and the obsessive compulsive disorder, schizophrenia, the unsuccessful suicide attempt, the thought of suicide or homicide are possessed or the past exists. 18)Anxiety is Gr 3 or more. 19)With mind abnormal conduction. Unstable angina Pericarditis Cardiac infarction Congestive heart failure Cardiomyopathy 20)LVFE is less than 50% 21)With abnormal cardiac conduction Serious Ventricular arrhythmias Supraventricular or tuberosity arrhythmia Abnormal cardiac conduction Other abnormal arrhythmia that uncontrolled with medicine 22)QT interval is extended, and administering the medicine to which the risk of inducing torusa-dodopoan has been received now. 23)Gastrointestinal functional disorder or digestive diseases with the possibility to influence the absorption remarkably are possessed. 24)Pt is unwilling or unable to comply with the protocol. 25)Pt is judged by the investigator. 26)Pregnancy or lactation
Target sample size	41

Research contact person
Name of lead principal investigator	Toshihiko Doi
Organization	National Cancer Center Hospital East
Division name	Department of Experimental Therapeutics,
Address	6-5-1, Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan
TEL	04-7133-1111
Email	bkm_core@east.ncc.go.jp

Public contact
Name of contact person	Takashi Kozima
Organization	National Cancer Center Hospital East
Division name	Gastrointestinal Oncology Division
Address	6-5-1, Kashiwanoha, Kashiwa, Chiba, 277-8577, Japan
TEL	04-7133-1111
Homepage URL
Email	bkm_core@east.ncc.go.jp

Sponsor
Institute	National Cancer Center Hospital East
Institute
Department

Funding Source
Organization	Novartis Pharmaceuticals
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	国立研究開発法人国立がん研究センター中央病院（東京都） 国立研究開発法人国立がん研究センター東病院（千葉県） 独立行政法人四国がんセンター(愛媛県） がん研究会有明病院(東京都） 埼玉県立がんセンター（埼玉） 愛知がんセンター中央病院（愛知県）

Other administrative information
Date of disclosure of the study information	2013 Year 07 Month 31 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2013 Year 05 Month 17 Day
Anticipated trial start date	2013 Year 07 Month 31 Day
Last follow-up date	2017 Year 03 Month 01 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2013 Year 07 Month 18 Day
Last modified on	2017 Year 03 Month 16 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013066