UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.
Title of the study (Brief title)	A phase I/II study of a WT1-W10 immunotherapy against high-risk MDS and MDS overt AML.
Region	Japan

Condition
Condition	High-risk MDS and MDS overt AML
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
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

Assessment
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

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	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

Eligibility
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

Research contact person
Name of lead principal investigator	Akihito Yokoyama
Organization	Kochi University, School of Medicine
Division name	Department of Hematology and respiratory Medicine
Address	Kohasu, Okocho, Nankoku, Kochi, 783-8505, Japan
TEL	088-866-5811
Email	vaccine@kochi-u.ac.jp

Public contact
Name of contact person	Keiko Udaka
Organization	School of Medicine, Kochi University
Division name	Anti-tumor Immunotherapy Research Network, Central Office, Department of Immunology
Address	Kohasu, Okocho, Nankoku, Kochi, 783-8505, Japan
TEL	088-880-2318
Homepage URL
Email	vaccine@kochi-u.ac.jp

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

Funding Source
Organization	NEC Corporation
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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 02 Month 07 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2009 Year 09 Month 03 Day
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

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2017 Year 02 Month 07 Day
Last modified on	2018 Year 02 Month 08 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000008125