UMIN-CTR Clinical Trial

Public title

A Phase I study of Glypican-3(GPC3) Peptide Vaccine for Pediatric Patients with Refractory Tumors

Acronym

GPC3 Vaccine PI for Pediatric Tumors

Scientific Title

A Phase I study of Glypican-3(GPC3) Peptide Vaccine for Pediatric Patients with Refractory Tumors

Scientific Title:Acronym

GPC3 Vaccine PI for Pediatric Tumors

Region

Japan

Condition

Refractory pediatric tumors

Classification by specialty

Pediatrics

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

To evaluate efficacy and safety of GPC3 peptide vaccine in pediatric patients with refractory malignant tumors

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Developmental phase

Phase I

Primary outcomes

Incidence of dose limiting toxicity

Key secondary outcomes

Progression free survival, overall survival etc.
GPC3-specific immune-responses to GPC3 vaccination

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

Vaccine

Interventions/Control_1

Intradermal injection of HLA-A24 or A2 restricted GPC3 peptide emulsified with Montanide ISA51 adjuvant, 1.5 mg/dose, repeating every 2 weeks, until disease progression/recurrence.

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

1

years-old

<=

Age-upper limit

20

years-old

>

Gender

Male and Female

Key inclusion criteria

1. Refractory pediatric tumors
1) Patients in refractory, recurrent, or progressive status (progressive group)
2) Patients in remission without chance of cure (remission group)
3) Patients in partial remission or stable disease (partial remission group)
2. Patients with histological confirmation of GPC3 expression in tumor cells
3. 1-19 year-old of age
4. ECOG performance status of 0-2
5. Confirming the following laboratory results within 14 days
1) Absolute neutrophil count >=1,000/mm3
2) Platelet count >= 75,000/mm3
3) Creatinine adjusted according to age as follows:
<= 0.8 mg/dl (<5 years)
<= 1.2 mg/dl (5 years-9 years)
<= 1.5 mg/dl (10 years-19 years)
4) Serum total Bilirubin <= 1.5mg/dl
and ALT <= 165 IU/l
6. More than 28 days since prior antitumor cytotoxic treatment
7. Sixteen to 19 year-old of age: written informed consent from both patient and legal guardian
Fifteen years and under: written informed consent from legal guardian
8. HLA-A24 or -A2 positive

Key exclusion criteria

1. Pleural effusion or ascites requiring removal by puncture
2. Active concurrent cancer or secondary cancer within 5 disease-free years of primary cancer
3. Active infection requiring systemic medication
4. Active gastrointestinal bleeding
5. Severe complications including cardiac failure, renal failure, liver failure, active gastro-duodenal ulcer, ileus, and uncontrolled diabetes mellitus
6. Severe psychiatric disorder
7. Past history of severe drug allergy
8. Systemic steroids or immunosuppressant medication
9. Judged inappropriate for the trial by a responsible researcher

Target sample size

15

Name of lead principal investigator

1st name
Middle name
Last name	Tetsuya Nakatsura

Organization

Research Center for Innovative Oncology, National Cancer Center Hospital East

Division name

Section for Cancer Immunotherapy

Zip code

Address

6-5-1, Kashiwanoha, Kashiwa, Japan

TEL

04-7131-5490

Email

tnakatsu@east.ncc.go.jp

Name of contact person

1st name
Middle name
Last name	Ako Hosono

Organization

National Cancer Center Hospital East

Division name

Department of Pediatric Oncology

Zip code

Address

6-5-1, Kashiwanoha, Kashiwa, Japan

TEL

04-7133-1111

Homepage URL

Email

ahosono@ncc.go.jp

Institute

Section for Cancer Immunotherapy,
Investigative Treatment Division, Research Center for Innovative Oncology, National Cancer Center Hospital East

Institute

Department

Personal name

Organization

The Ministry of Health, Labor and Welfare, Japan

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Japan

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

Date of disclosure of the study information

2011

Year

09

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

No longer recruiting

Date of protocol fixation

2011

Year

02

Month

03

Day

Date of IRB

Anticipated trial start date

2011

Year

09

Month

01

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2011

Year

09

Month

15

Day

Last modified on

2016

Year

03

Month

15

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000007502