UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase I Dose-escalation, Safety / Tolerability and Preliminary Efficacy Study of Intratumoral Administration of GEN0101 in Patients with Advanced Melanoma.
Title of the study (Brief title)	Phase I study of GEN0101 in Patients with Advanced Melanoma.
Region	Japan

Condition
Condition	Advanced Melanoma
Classification by specialty	Dermatology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Assessing the Safety and Tolerability of GEN0101
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase I

Assessment
Primary outcomes	Assessing the Safety and Tolerability of GEN0101
Key secondary outcomes	Assessing the antitumor immunity and validity of GEN0101

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	open-label ,multicenter trial Patients receive GEN0101 (30,000m NAU/day or 60,000 mNAU/day) intratumorally 2 triweekly every 4 weeks (week 1 and 2). One such cycle is repeated 2 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	86 years-old >
Gender	Male and Female
Key inclusion criteria	1) The patient is Able to understand and sign the Informed Consent Document. 2) The patient is at least more than 20 years and less than 86 years old. 3) The patient has a diagnosis of malignant tumor as confirmed by histopathology or cytology. 4) The patient has a diagnosis of malignant melanoma progressive melanoma in AJCC stage IIIC or stage IV who meets either below condition; Recurrent Refractory or inapplicable to a standard treatment The patient refuses a standard treatment. 5) The patient has measurable or evaluable superficial skin lesions, 10 mm or larger (more than 3 sites and each site must be more than 3mm). 6) The patient has a life expectancy for at least 8 weeks or more. 7) The patient has a ECOG Performance Status Scale of 0 or 2. 8) The organs function adequately evaluated by the following lab values; WBC: equal or more than 2,000/uL PLT: equal or more than 75,000/uL Hemoglobin: equal or more than 8.0 g/dL AST:<local reference range 2.5 ALT:<local reference range 2.5 T-Bil:<local reference range 2 Serum creatinine:<local reference range 2
Key exclusion criteria	1) The patient has multiple brain metastases. 2) The patient shows positive immune response by GEN0101 prick test at screening. 3) The patient has a uncontrolled serious complication such as active infection. 4) Chemotherapy, radiotherapy, local IFN-b therapy, and/or any other therapy with established or suggested anti-cancer effects within 4 weeks (in case of Nitrosoureas or Mitomycin C: within 6 not 4 weeks). 5) Participation in another clinical trial of another investigational medical product within 4 weeks. 6) History of a second independent malignancy within 5 years. 7) History of active autoimmune disease 8) The patient is undergoing on systemic corticosteroids or immunosuppressive agents except for the use of maximum 10 mg/day oral prednisolone over 6 months. 9) The patient is a pregnant or lactating female. 10) The patient has any serious psychiatric disorders that may be unwilling or unable to comply with protocol requirements and scheduled visits. 11) The patient has a history of a transplantation of the allogeneic ogan, the autologous ogan or tissue. 12) The patient shows adequate blood property with PT and APTT more than 1.5 times of the upper limit of local reference range. 13) The patient has hepatitis Be antigen, hepatitis C antibody or human immunodeficiency virus (HIV1,2) antibody positive status. 14) The Patient is inappropriate to be enrolled in this study judged by the doctors in charge.
Target sample size	15

Research contact person
Name of lead principal investigator	Ichiro Katayama
Organization	Osaka University Hospital
Division name	Dermatology
Address	2-15 Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6879-5111
Email	katayama@derma.med.osaka-u.ac.jp

Public contact
Name of contact person	Atsushi Tanemura
Organization	Osaka University Hospital
Division name	Dermatology
Address	2-15 Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6879-5111
Homepage URL
Email	tanemura8@hotmail.co.jp

Sponsor
Institute	Department of Dermatology Course of Integrated Medicine Graduate School of Medicine, Osaka University
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and development
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

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	大阪大学医学部附属病院（大阪府）、国立がん研究センター中央病院（東京都）、静岡県立静岡がんセンター（静岡県）

Other administrative information
Date of disclosure of the study information	2014 Year 03 Month 01 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2013 Year 12 Month 25 Day
Anticipated trial start date	2014 Year 11 Month 07 Day
Last follow-up date	2016 Year 05 Month 02 Day
Date of closure to data entry	2016 Year 06 Month 29 Day
Date trial data considered complete	2016 Year 06 Month 29 Day
Date analysis concluded	2016 Year 08 Month 31 Day

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

Management information
Registered date	2014 Year 01 Month 24 Day
Last modified on	2017 Year 01 Month 17 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000015136