UMIN-CTR Clinical Trial

Public title

Phase 1/2 clinical trial of immunotherapy with fusions of tumor cells and dendritic cells for pediatric refractory brain tumor

Acronym

Dendritic cell-based immunotherapy for pediatric refractory brain tumor

Scientific Title

Phase 1/2 clinical trial of immunotherapy with fusions of tumor cells and dendritic cells for pediatric refractory brain tumor

Scientific Title:Acronym

Dendritic cell-based immunotherapy for pediatric refractory brain tumor

Region

Japan

Condition

Pediatric refractory brain tumor

Classification by specialty

Pediatrics

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

To evaluate the safety and efficacy of immunotherapy with fusion cells of tumor cells and dendritic cells for refractory brain tumor in childhood and AYA.

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Pragmatic

Developmental phase

Phase I,II

Primary outcomes

Safety

Key secondary outcomes

2-year overall survival (OS) rate, 1-year progression free survival (PFS) rate, iRANO criteria

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Historical

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

1

Purpose of intervention

Treatment

Type of intervention

Vaccine

Interventions/Control_1

In Phase 1, we evaluate the safety (DLT) by three steps according to 3+3 design method. In Phase 2, we evaluate the safety and efficacy with 0.5-10 x10^6 cells in dendritic cell count. Fusion cell inoculation will be repeated 3-10 times in each 28-84 days cycle.

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

3

years-old

<=

Age-upper limit

25

years-old

>

Gender

Male and Female

Key inclusion criteria

<Tentative inclusion criteria>
Patients will be tentatively enrolled in this clinical trial, when they satisfy the following tentative inclusion criteria.

1) A patient who is suspected a relapse or refractory pediatric brain tumor with poor prognosis.
2) A patient who needs to receive a surgical tumor resection.
3) A patient who is tolerable to receive gathering peripheral blood mononuclear cells by an apheresis method.
4) Age at enrollment is 3 to <25 year-old
5) A patient who has grade 0 to 3 of the Performance Status. Patients with a PS score of 4 due to neurological symptoms associated with the primary disease may be eligible if patients could go to outpatient clinic without any respiratory supports.
6) A patient who is suspected to be alive for more than 3 months.
7) A patient who does not have multiple primary cancer.
8) A patient for whom either the legally acceptable representative or the patient (if aged > 16 years) have provided written voluntary consent to participation in this study after fully receiving and understanding the information about this study.
9) A patient who satisfies following criteria without functional failure of major organs, such as heart, lung, liver, kidney, and bone marrow etc.
WBC > 1,500/microL
Platelet > 50,000/microL
Hb > 9.0/dL
Serum creatinine < 3-fold the upper limit of the normal range based on each age (ULN)
Total bilirubin < 3-fold the ULN
AST, ALT < 120U/L

<Final inclusion criteria>
Tentatively enrolled patients will be truly enrolled as a subject of this clinical trial, when they satisfy the following final inclusion criteria.

1) A patient who is histologically proven recurrent or refractory brain tumor.
2) A patient who has autologous cultured tumor cells available to fuse with dendritic cells.

Key exclusion criteria

1) A patient who has sever immune deficiency.
2) A patient who has a past or current history of autoimmune disease.
3) A patient who is administrated immunosuppressive agents for a long period, and is difficult to stop it.
4) A patient who received chemotherapy (including with molecular target therapy) or radiotherapy within 21 days.
5) A patients who underwent allogeneic hematopoietic stem cell transplant.
6) A patient who had recovered from some viral infection (influenza virus or gastroenteritis) within 14 days.
7) A patient who has an allergy to biological medicines.
8) A patient who previously received WT1 peptide vaccine or other immunotherapy.
9) A patient who has positive of HBs antigen, HIV antibody, HCV antibody, or HTLV antibody.
10) A patient who has severe infectious disease over Grade 3 of CTCAE v4.0.
11) A patient who has sever lung disease (Class 3 or more in the Hugh-Jones classification).
12) A patient who has severe heart disease (Class 3 or more in the NYHA).
13) A patient who has some uncontrollable complication.
14) A pregnant or lactating patient.
15) A patient who is involved in other clinical trials.
16) A patients who are inappropriate for participation in the study for other reasons in the opinion of the investigator or sub-investigator.

Target sample size

20

Name of lead principal investigator

1st name
Middle name
Last name	Takaaki Yanagisawa

Organization

Jikei University School of Medicine

Division name

Neurosurgery

Zip code

Address

3-25-8 Nishishinbashi Minato-ku, Tokyo

TEL

81-3-3433-1111

Email

takaakipno@jikei.ac.jp

Name of contact person

1st name
Middle name
Last name	Masayoshi Yamaoka

Organization

Jikei University School of Medicine

Division name

Pediatrics

Zip code

Address

3-25-8 Nishishinbashi Minato-ku, Tokyo

TEL

81-3-3433-1111

Homepage URL

Email

yamaoka@jikei.ac.jp

Institute

Jikei University School of Medicine

Institute

Department

Personal name

Organization

Jikei University School of Medicine

Organization

Division

Category of Funding Organization

Self funding

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

東京慈恵会医科大学附属病院

Date of disclosure of the study information

2018

Year

02

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

2018

Year

02

Month

08

Day

Date of IRB

Anticipated trial start date

2018

Year

02

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

2018

Year

02

Month

15

Day

Last modified on

2018

Year

02

Month

15

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000035722