UMIN-CTR Clinical Trial

Public title

Phase 1 Dose-escalation, Safety / Tolerability and Preliminary Efficacy Study of Intratumoral and Subcutaneous Administration of GEN0101 in Patients with Chemotheapy-resistant Malignant Pleural Mesothelioma (Phase I).

Acronym

Phase 1 study of GeN0101 in patients suffering from Chemotheapy-resistant Malignant Pleural Mesothelioma

Scientific Title

Phase 1 Dose-escalation, Safety / Tolerability and Preliminary Efficacy Study of Intratumoral and Subcutaneous Administration of GEN0101 in Patients with Chemotheapy-resistant Malignant Pleural Mesothelioma (Phase I).

Scientific Title:Acronym

Phase 1 study of GeN0101 in patients suffering from Chemotheapy-resistant Malignant Pleural Mesothelioma

Region

Japan

Condition

Chemotheapy-resistant Malignant Pleural Mesothelioma

Classification by specialty

Chest surgery

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

Primary endopoint:To determine the recommended dosage foe the phase 2 study through the assessment of DLT(Dose Limiting Toxicity)

Secondary endopoint: to evaluate the preliminary efficacy

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Others

Trial characteristics_2

Developmental phase

Phase I

Primary outcomes

Assessment of DLT

Key secondary outcomes

1)Effect of tumor shirinkage
- RECIST
- Tumor marker (SMRP, CYFRA)
2)Induction of antitumor immunity
- NK cell
- IL-6
- IFN-gammma
3)Blood concentration of GEN0101/HVJ-E

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

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

Age-lower limit

20

years-old

<=

Age-upper limit

85

years-old

>=

Gender

Male and Female

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 pleural mesothelioma as confirmed by histology
4) Patinets suffering from chemoresistant malignant pleural mesothelioma, or patients without consecutive chemotherapy.
- More than 6 week between the end date of the Chemotherapy and the registration date when the Chemotherapy has been ineffective
5) PaO2 >=70 mmHg and SpO2>=93%
6) Expected survival period is more than 8 weeks after planned start date of investigational product
7) ECOG Performance Status 0 or 1
8) Patients with lesions which can be detected by diagnostic imaging such as MRI or CT scan prior to treatment, and which can be administered with GEN0101/HVJE
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, radiotherapy or immunotherapy within 6 weeks before planned registration date
5) Received another investigational product within 4 weeks before the informed concent
6) Had a history of othr malignancy, except for the relapse-free and metastasis-free for more than 5 years after the last treatment at the registration
7) Have a interstitial pneumonia or fibrosing disease of the lung that need treatment
8) Have an active autoimmune disease
9)Receiving systemic administration of glucocorticosteroid which restrains immunity response.
10) Had a history of the autologous or homogeneous organ or tissue transplantation (Receiving immunosuppressive medication)
11) 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
12) Pregnant or lactating women, except for the woman who discontinue to lactate (from the day of a written informed consent to the day after 30days of discontinue the administration)
In the case of womane, to conduct beta-HCG tests to confirm the presenceor absence of pregnancy
13) Positive result of the hepatitis B surface antigen, HCV antibody or HIV test at the screening visit
14) Inappropriate to be enrolled in this study judged by the investigators

Target sample size

12

Name of lead principal investigator

1st name
Middle name
Last name	Meinoshin Okumura

Organization

Osaka University Hospital

Division name

Center for respiratory

Zip code

Address

2-15 Yamadaoka, Suita-shi, Osaka 565-0871, Japan

TEL

06-6879-3152

Email

meinosin@thoracic.med.osaka-u.ac.jp

Name of contact person

1st name
Middle name
Last name	Katsuhisa Saito

Organization

Osaka University Hospital

Division name

Medical Center for Translational and Clinical Research

Zip code

Address

2-2 Yamadaoka, Suita-shi, Osaka 565-0871, Japan

TEL

06-6210-8289

Homepage URL

Email

saitokt@dmi.med.osaka-u.ac.jp

Institute

Osaka University Hospital, Center for respiratory

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Other

Nationality of Funding Organization

Co-sponsor

GenomIdea,Inc.

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

Date of disclosure of the study information

2015

Year

10

Month

15

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

Open public recruiting

Date of protocol fixation

2015

Year

09

Month

17

Day

Date of IRB

Anticipated trial start date

2015

Year

11

Month

15

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2015

Year

10

Month

14

Day

Last modified on

2015

Year

10

Month

14

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022270