UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A study to expand access to vemurafenib for patients with BRAFV600 mutation-positive unresectable/recurrent melanoma.
Title of the study (Brief title)	Vemurafenib(Expand Access Program)
Region	Japan

Condition
Condition	melanoma
Classification by specialty	Dermatology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To provide vemurafenib to patients with BRAFV600 mutation-positive unresectable/recurrent melanoma for whom few therapeutic options are available.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Safety Outcome Measures # Incidence, type, and severity of AEs # Incidence and nature of serious adverse events (SAEs) # Incidence of AEs leading to vemurafenib discontinuation or interruption # Cause of death on study
Key secondary outcomes

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	Enrolled patients will receive Vemurafenib at 960mg p.o. BID.
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)Patients who have been fully informed about the study and have given written consent themselves. (2)Patients aged 20 years or older at the time of consent. (3) Eastern Cooperative Oncology Group (ECOG) Performance Status of 0,1 or 2 (4)Patients with evidence of the presence of BRAFV600 mutation in tumor tissue. (5)Patients who have no treatment history of unresectable/recurrent melanoma or who have received at least one regimen for melanoma and had disease progression documented during or after treatment. (6)Patients who have adequately recovered from the toxicity of the most recent systemic or local therapy for unresectable/recurrent melanoma. (7)Adequate organ function as assessed by the following clinical laboratory parameters within 28 days prior to initiation of treatment:(Assessments conducted 4 weeks prior to the first dose of study drug [i.e., the same day of the previous week] will be allowed.) 1)ANC; 1500/mm3=< 2)Platelets; 100,000/mm3=< 3)Hemoglobin; 9.0 g/dL=< 4)Serum Creatinine; 1.5x upper limit of normal, or creatinine clearance > 50 mL/min 5)Serum direct Bilirubin; 1.5x upper limit of normal or; 5x ULN if concurrent liver metastases. 6)Serum AST and ALT; 2.5x upper limit of normal or;5x ULN if concurrent liver metastases. (8)For women of childbearing potential, negative serum or urine pregnancy test within 7 days prior to treatment initiation. (9)For women of childbearing potential and men who are partners of women of childbearing potential, willingness to practice effective contraception during the study and for 6 months after treatment discontinuation.
Key exclusion criteria	(1)Patients with brain metastases However, they will be eligible if they have only asymptomatic brain metastases treated by surgical resection or stereotaxic radiation therapy and diagnosed by the investigator/subinvestigators as being free of symptoms, with imaging evidence of stable disease. (2)Patients with concurrent malignancies with a disease-free period of less than 2 years, except cutaneous basal cell carcinoma, cutaneous squamous cell carcinoma, cervical carcinoma in situ, and metastatic prostate cancer that has increased prostate-specific antigen (PSA) levels but is not clinically diagnosed by imaging (3)Patients who are pregnant or nursing (4)Patients with difficulty taking oral medications (5)Patients who require concomitant use of other anticancer treatments (e.g., chemotherapy, other molecular-targeted agents, radiation therapy, and other investigational or experimental drugs) (6)Patients who have had the following medical conditions within 6 months prior to the first dose of study drug: Myocardial infarction, severe or unstable angina pectoris, symptomatic congestive heart failure, cerebrovascular disease or transient ischemic attack, pulmonary embolism, and inadequately controlled hypertension (7)Patients with a mean corrected QT (QTc) interval of 450 msec or longer at screening (8)Patients with Grade 2 (as defined by the National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] Version 4.0) or greater arrhythmia (9)Patients with inadequately controlled concurrent medical conditions (e.g., those requiring intravenous antibiotic therapy) (10)Patients who are unable to use acceptable methods of contraception (11)Patients who are, in the opinion of the investigator/subinvestigators, ineligible for participation in the study for any other reason
Target sample size	10

Research contact person
Name of lead principal investigator	Naoya Yamazaki
Organization	National Cancer Center Hospital
Division name	Dermatology
Address	5-1-1, Tsukiji, Chuo-ku, Tokyo, 104-0045, Japan
TEL	03-3542-2511
Email	vem_core@east.ncc.go.jp

Public contact
Name of contact person	Naoya Yamazaki
Organization	National Cancer Center Hospital
Division name	Dermatology
Address	5-1-1, Tsukiji, Chuo-ku,Tokyo,104-0045,Japan
TEL	03-3542-2511
Homepage URL
Email	vem_core@east.ncc.go.jp

Sponsor
Institute	National Cancer Center Hospital East
Institute
Department

Funding Source
Organization	Vemurafenib are provided gratis by Chugai pharm Co., Ltd. It is adopted as grant of expand access pilot program by Ministry of Health, Labour and Welfare.
Organization
Division
Category of Funding 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 国立研究開発法人　国立がん研究センター東病院

Other administrative information
Date of disclosure of the study information	2014 Year 11 Month 01 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2014 Year 09 Month 04 Day
Anticipated trial start date	2014 Year 11 Month 10 Day
Last follow-up date
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	2014 Year 10 Month 31 Day
Last modified on	2016 Year 12 Month 13 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017901