UMIN-CTR Clinical Trial

Public title

A study to expand access to vemurafenib for patients with BRAFV600 mutation-positive unresectable/recurrent melanoma.

Acronym

Vemurafenib(Expand Access Program)

Scientific Title

A study to expand access to vemurafenib for patients with BRAFV600 mutation-positive unresectable/recurrent melanoma.

Scientific Title:Acronym

Vemurafenib(Expand Access Program)

Region

Japan

Condition

melanoma

Classification by specialty

Dermatology

Classification by malignancy

Malignancy

Genomic information

NO

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

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

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

Name of lead principal investigator

1st name
Middle name
Last name	Naoya Yamazaki

Organization

National Cancer Center Hospital

Division name

Dermatology

Zip code

Address

5-1-1, Tsukiji, Chuo-ku, Tokyo, 104-0045, Japan

TEL

03-3542-2511

Email

vem_core@east.ncc.go.jp

Name of contact person

1st name
Middle name
Last name	Naoya Yamazaki

Organization

National Cancer Center Hospital

Division name

Dermatology

Zip code

Address

5-1-1, Tsukiji, Chuo-ku,Tokyo,104-0045,Japan

TEL

03-3542-2511

Homepage URL

Email

vem_core@east.ncc.go.jp

Institute

National Cancer Center Hospital East

Institute

Department

Personal name

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

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

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

Date of disclosure of the study information

2014

Year

11

Month

01

Day

URL releasing protocol

Publication of results

Unpublished

URL related to results and publications

Number of participants that the trial has enrolled

Results

Results date posted

Results Delayed

Results Delay Reason

Date of the first journal publication of results

Baseline Characteristics

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

Completed

Date of protocol fixation

2014

Year

09

Month

04

Day

Date of IRB

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

Other related information

Registered date

2014

Year

10

Month

31

Day

Last modified on

2016

Year

12

Month

13

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017901