UMIN-CTR Clinical Trial

Public title

A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.

Acronym

A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.

Scientific Title

A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.

Scientific Title:Acronym

A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.

Region

Japan

Condition

High-risk MDS and MDS overt AML

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

The aim of the study is to examine the safety and efficacy of an immunotherapy using WT1-W10 peptide and pertussis whole cell-vaccine. The target diseases are MDS and MDS overt AML. W10 peptide is presented by HLA-A*24:02, A*02:01, A*02:06, A*02:07, thus the patients bearing one or two of these alleles are eligible to the study.

Basic objectives2

Safety,Efficacy

Basic objectives -Others

Trial characteristics_1

Trial characteristics_2

Developmental phase

Phase I,II

Primary outcomes

Phase I : adverse events of grade 3 or higher, all adverse events by the CTCAE criteria
Phase II : progression free survival

Key secondary outcomes

Recurrence rate, survival rate, overall survival, maximal response, specific immune responses

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

Vaccine

Interventions/Control_1

3.0mg of WT1 peptide and adjuvant agent well be administrated intradermally. The administration interval is essentially every week.

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 diagnosed as MDS by the WHO criteria. Among them the high-risk and very high risk groups by the WHO prognosis scoring system are recruited.
2) patients who have been informed of the disease
3) patients who have no option to standard therapies or those who chose this trial over the standard therapy. Patients are not eligible to this trial if treated with the previous therapy within 4 weeks.
4) patients bearing at least one of HLA-A*24:02, A*02:01, A*02:06, A*02:07 genes.
5) Overexpression of WT1 gene was observed by real-time PCR at least once in the bone marrow or peripheral blood.
normal threshold of the WT1 transcripts:
in bone marrow or peripheral blood: =< 250 copy/ug RNA
Alternatively, over-expression of WT1 protein is confirmed by flow cytometry.
6) The presence of residual tumors in the bone marrow or peripheral blood are confirmed by one of the clinical tests listed below.
The presence of leukemic blasts
Overexpression of the WT1 transcript
The presence of canonical chromosomal abnormalities have been confirmed by chromosome examination, FISH or analysis of chimeric transcripts.
7) At least 8 days have passed after the administration of either hematopoietic factors, transfusion of platelets or RBC.
blasts in the bone marrow and peripheral blood < 50%,
neutrophil >= 500 /ul
platelet >= 20,000 /ul
Hb >= 6.5 g/dl
8) No involvement of the central nervous system or under control
9) 20 years of age or older, and less than 85 years
10) The performance status should be between 0-1 by the ECOG criteria
11) Functions of the major organs are preserved.
12) No serious complications, No double tumors including hematopoietic malignancy.
13) Written consent have been obtained from patients.

Key exclusion criteria

1)patients with infectious diseases including active Tuberculosis which are poorly controlled.
2)patients with serious comorbidities (generally those with grade 3 or higher by the NCI-CTC criteria ver 3.0)
3)pregnant women, Breast feeding mothers
4)patients with severe mental problems.
5)patients who have already been recruited in other clinical trials.
6) Patients who have dropped out after starting this clinical trials.

Target sample size

60

Name of lead principal investigator

1st name
Middle name
Last name	Akihito Yokoyama

Organization

Kochi University, School of Medicine

Division name

Department of Hematology and respiratory Medicine

Zip code

Address

Kohasu, Okocho, Nankoku, Kochi, 783-8505, Japan

TEL

088-866-5811

Email

vaccine@kochi-u.ac.jp

Name of contact person

1st name
Middle name
Last name	Keiko Udaka

Organization

School of Medicine, Kochi University

Division name

Anti-tumor Immunotherapy Research Network, Central Office, Department of Immunology

Zip code

Address

Kohasu, Okocho, Nankoku, Kochi, 783-8505, Japan

TEL

088-880-2318

Homepage URL

Email

vaccine@kochi-u.ac.jp

Institute

Department of Hematology and respiratory Medicine, School of Medicine, Kochi University

Institute

Department

Personal name

Organization

NEC Corporation

Organization

Division

Category of Funding Organization

Profit organization

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

Date of disclosure of the study information

2017

Year

02

Month

07

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

Completed

Date of protocol fixation

2009

Year

09

Month

03

Day

Date of IRB

Anticipated trial start date

2009

Year

09

Month

30

Day

Last follow-up date

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Registered date

2017

Year

02

Month

07

Day

Last modified on

2018

Year

02

Month

08

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000008125