UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase 1 dose-escalation, safety / tolerability and preliminary efficacy study of intratumoral and subcutaneous administration of GEN0101 in patients with recurrence of castration resistant prostate cancer
Title of the study (Brief title)	Phase 1 study of GEN0101 in patients with recurrence of CRPC
Region	Japan

Condition
Condition	Castration resistant prostate cancer
Classification by specialty	Urology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Primary endpoint: To determine the recommended dosage for the phase 2 study through the assessment of DLT(Dose Limiting Toxicity) Secondary endpoint: to evaluate the preliminary efficacy
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Others
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Assessment of DLT
Key secondary outcomes	1) Effect of tumor shrinkage - RECIST - Tumor marker (PSA: Prostate Specific Antigen, NSE: Neuron-specific enolase, CEA: Carcinoembryonic Antigen, CA19-9: Carbohydrate Antigen19-9) - histological evaluation 2) Induction of antitumor immunity - NK cell activity - IL-6 - IFN-gamma

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	GEN0101 will be administered four times per two weeks and then washed out for two weeks. This one cycle will be repeated twice. GEN0101 will be given at a dose of 30,000mNAU for each injection. If dose-escalation is permitted by independent data monitoring committee, another cohort will be given at a dose of 60,000mNAU for each injection.
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	85 years-old >=
Gender	Male
Key inclusion criteria	1) Patients providing a written informed consent by voluntary agreement. 2) Age 20 =< and =<85 years old at the time of informed consent 3) Have a diagnosis of malignant tumor as confirmed by histology or cytology. 4) Have a diagnosis of recurrence of castration resistant prostate cancer patient and meet below the following condition - Inapplicable to the standard treatment, ineffective through the criteria of the Prostate Cancer Clinical Trials Working Group (PCWG2) or refuse the standard treatment - More than 3 weeks between the end date of the standard treatment and the registration date when the standard treatment has been ineffective 5) Serum PSA <100 ng/mL at the screening visit 6) Expected survival period is more than 8 weeks after planned start date of investigational product 7) ECOG Performance Status 0 or 1 8) Have an injectable intraprostatic lesion confirmed by histologic examination 9) The marrow function, liver function and the kidney function must be kept as follows at the screening visit (1) leukocyte >= 3,000/mcL (2) neutrophil >=1,500/mcL (3) platelet >=75,000/mcL (4) hemoglobin >=8.0 g/dL. (5) AST =<100 IU/L (6) ALT =<100 IU/L (7) total bilirubin =<2.5 mg/dL (8) serum creatinine =<2.5 mg/dL
Key exclusion criteria	1) Have multiple brain metastases 2) Positive result of the prick test of GEN0101 3) Have serious complications such as uncontrolled active infection 4) Received systemic chemotherapy within 3 weeks before planned registration date or received radiotherapy or immunotherapy within 6 weeks before planned registration date /However the hormone therapy except for the estramustine, enzalutamide and abiraterone, bisphosphonate and anti-RANKL antigen antibody are not included in the systemic chemotherapy. 5) Received another investigational product within 4 weeks before the informed concent 6) Had a history of malignancy other than prostate cancer, except for the relapse-free and metastatis-free for more than 5 years after the last treatment at the registration 7) Have an active autoimmune disease 8)Receiving systemic administration of glucocorticosteroid which restrains immunity response, except for the administration for a long period (over 6 months) of the low dose (equivalent to under 10 mg/day oral prednisolone). 9) Had a history of the autologous or homogeneous organ or tissue transplantation (Receiving immunosuppressive medication) 10) PT(%) less than 10% of the lower limit of normal or APTT more than 1.5 times of the upper limit of normal of local reference range at the screening visit 11) Positive result of the hepatitis B surface antigen, HCV antibody or HIV test at the screening visit 12) Inappropriate to be enrolled in this study judged by the investigators
Target sample size	12

Research contact person
Name of lead principal investigator	Norio Nonomura
Organization	Osaka University Hospital
Division name	Department of Urology
Address	2-15 Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6879-3531
Email	nono@uro.med.osaka-u.ac.jp

Public contact
Name of contact person	Katsuhisa Saito
Organization	Osaka University Hospital
Division name	Medical Center for Translational Research
Address	2-2 Yamadaoka, Suita-shi, Osaka 565-0871, Japan
TEL	06-6210-8289
Homepage URL
Email	saitokt@dmi.med.osaka-u.ac.jp

Sponsor
Institute	Osaka University
Institute
Department

Funding Source
Organization	Japan Science and Technology Agency
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	ISHIHARA SANGYO KAISHA,LTD. GenomIdea,Inc.
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）　　　　　　　　Osaka University Hospital（Osaka）

Other administrative information
Date of disclosure of the study information	2015 Year 04 Month 15 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 03 Month 03 Day
Anticipated trial start date	2015 Year 06 Month 12 Day
Last follow-up date	2017 Year 08 Month 22 Day
Date of closure to data entry	2017 Year 09 Month 27 Day
Date trial data considered complete	2017 Year 10 Month 26 Day
Date analysis concluded	2017 Year 11 Month 22 Day

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

Management information
Registered date	2015 Year 04 Month 09 Day
Last modified on	2017 Year 10 Month 18 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019824