UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000027867
Receipt number R000029432
Scientific Title Phase 1 clinical trial of WT1 peptide vaccine therapy for pediatric cancers.
Date of disclosure of the study information 2017/06/26
Last modified on 2023/12/27 18:52:46

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Basic information

Public title

Phase 1 clinical trial of WT1 peptide vaccine therapy for pediatric cancers.

Acronym

Phase 1 clinical trial of WT1 peptide vaccine therapy for pediatric cancers.

Scientific Title

Phase 1 clinical trial of WT1 peptide vaccine therapy for pediatric cancers.

Scientific Title:Acronym

Phase 1 clinical trial of WT1 peptide vaccine therapy for pediatric cancers.

Region

Japan


Condition

Condition

Pediatric refractory solid tumors or leukemia

Classification by specialty

Pediatrics

Classification by malignancy

Malignancy

Genomic information

NO


Objectives

Narrative objectives1

To assess the safety and feasibility of WT1 peptide vaccination with adjuvant OK-432.

Basic objectives2

Safety

Basic objectives -Others


Trial characteristics_1

Exploratory

Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

Safety

Key secondary outcomes

Overall survival, The ratio of the patients who completed the course, WT1 specific CTL rate in peripheral blood mononuclear cells (PBMCs), Lymphocyte subsets, WT1-specific interferon-gamma production of PBMCs


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

Vaccine

Interventions/Control_1

Seven doses of HLA-A*24- or A*02-restricted WT1 peptide (1-3mg) emulsified with 0.5-2KE of adjuvant will be intradermally injected on bilateral axillary and inguinal areas every 2 weeks. WT1 peptide dose will be adjusted according to patient's body weight (1mg of WT1 peptide for body weight (BW) <10kg, 2mg for 10kg < BW <20kg, 3mg for BW >30kg). Injection schedule can be changed within 3 days.

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

1 years-old <=

Age-upper limit

20 years-old >

Gender

Male and Female

Key inclusion criteria

1. Patients with pathologically proven pediatric cancers.
2. Patients treated with operation, chemotherapy, and radiotherapy accoding to standard clinical guidelines.
3. Patients who did not received any specific immunotherapy before enrollement of this trial.
4. Patients with HLA-A*02 or A*24 and having neither allergies to OK-432 nor to penicillin.
5. Patients aged 19 years or under at the time of informed concent.
6. Karnofsky performance Status (KPS) are over 60%.
7. Estimated survival duration is over 3 months.
8. Function of major organs is maintained, with the following inclusion criteria.
A) Leukocyte count>=3,000/mm3
B) Lymphocyte count>=500/ mm3
C) Platelet count>=100,000/mm3
D) Hemoglobin concentration >=10.0g/dL
E) AST/ALT <2.5x ULN
F) Serum creatinine <= 1.5xULN
G) APTT/PT-INR <= 1.5xULN

Key exclusion criteria

1. Patients with severe organ dysfunction, infection under treatment, blood abonormalities or bleeding tendency.
2. Patients with leukemia or lymphoma are excluded.

Target sample size

15


Research contact person

Name of lead principal investigator

1st name Kazuo
Middle name
Last name Sakashita

Organization

Nagano Children's Hospital

Division name

Department of Hematology and Oncology

Zip code

399-8288

Address

3100 Toyoshina Azumino city, Nagano

TEL

0263-73-6700

Email

sakasita@shinshu-u.ac.jp


Public contact

Name of contact person

1st name Sakashita
Middle name
Last name Kazuo

Organization

Nagano Children's Hospital

Division name

Department of Hematology and Oncology

Zip code

399-8288

Address

3100 Toyoshina Azumino city, Nagano

TEL

0263-73-6700

Homepage URL


Email

sakasita@shinshu-u.ac.jp


Sponsor or person

Institute

Nagano Children's Hospital

Institute

Department

Personal name



Funding Source

Organization

Nagano Children's Hospital

Organization

Division

Category of Funding Organization

Self funding

Nationality of Funding Organization



Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization

Nagano Children's Hospital

Address

3100 Toyoshina Azumino City, Nagano Prefecture

Tel

0263-73-6700

Email

kodomo@pref-nagano-hosp.jp


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



Other administrative information

Date of disclosure of the study information

2017 Year 06 Month 26 Day


Related information

URL releasing protocol

https://rctportal.niph.go.jp/s/detail/um?trial_id=UMIN000027867

Publication of results

Unpublished


Result

URL related to results and publications

none

Number of participants that the trial has enrolled

10

Results

Of the 24 patients, 18 completed the scheduled vaccinations. Sixteen patients had local skin symptoms and/or fever. In 1 patient, anaphylactic symptoms emerged at the time of the final injection, but these quickly subsided after the treatment. WT1-specific immunological responses were observed in 4 patients (22.2%). WT1 and HLA class I expression were confirmed in 100% and 85% of primary tumors, respectively. WT1 peptide vaccine therapy combined with OK-432 appears to be relatively safe for children.

Results date posted

2023 Year 06 Month 28 Day

Results Delayed


Results Delay Reason


Date of the first journal publication of results


Baseline Characteristics

Patients with pediatric cancers (Aged from 1 to 19 years)
1. Patients with severe organ dysfunction, infection under treatment, blood abnormalities or bleeding tendency.
2. Patients with leukemia or lymphoma are excluded.

Participant flow

Paediatric patients with a solid tumor were vaccinated with a WT1 peptide and OK-432 once every 2 weeks, for a total of seven times.

Adverse events

Sixteen patients had local skin symptoms and/or fever. In 1 patient, anaphylactic symptoms emerged at the time of the final injection, but these quickly subsided after the treatment.

Outcome measures

Primary outcome: Safety
Secondary outcome: Overall survival, The ratio of the patients who completed the course, WT1-specific CTL rate in peripheral blood mononuclear cells, Lymphocyte subsets, WT1-specific interferon-gamma production of PBMCs

Plan to share IPD


IPD sharing Plan description



Progress

Recruitment status

Completed

Date of protocol fixation

2017 Year 01 Month 01 Day

Date of IRB

2017 Year 01 Month 01 Day

Anticipated trial start date

2017 Year 06 Month 27 Day

Last follow-up date

2019 Year 03 Month 30 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information



Management information

Registered date

2017 Year 06 Month 22 Day

Last modified on

2023 Year 12 Month 27 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029432


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name