UMIN-CTR Clinical Trial

Basic information
Public title	Phase I study of WT1-expressing human artificial adjuvant vector cells (aAVC-WT1) for patients with relapsed or refractory acute myeloid leukemia
Acronym	Phase I study of WT1-expressing human artificial adjuvant vector cells (aAVC-WT1) for patients with relapsed or refractory acute myeloid leukemia
Scientific Title	Phase I study of WT1-expressing human artificial adjuvant vector cells (aAVC-WT1) for patients with relapsed or refractory acute myeloid leukemia
Scientific Title:Acronym	Phase I study of WT1-expressing human artificial adjuvant vector cells (aAVC-WT1) for patients with relapsed or refractory acute myeloid leukemia
Region	Japan

Condition
Condition	relapsed or refractory acute myeloid leukemia (AML)
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To explore the safety of aAVC-WT1, and secondaly to explore immunological effects (NKT cell-specific immune response) and clinical efficacy
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Safety (1) Occurence of Dose Limiting Toxicity and determination of Maximum Tolerated Dose (2) Exploration of adverse events (3) Statistical analysis on examinations related to safety evaluation
Key secondary outcomes	Immunological effects (NKT cell-specific immune response and amplification rate) Clinical efficacy (1) Hematological effect: Response rate(CR, PR, CRi) (2) Longitudinal change of WT1 mRNA copies (3) Overall survival

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	Intravenous administraion of aAVC-WT1 followed by 3+3 dose escalation scheme
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	Not applicable
Gender	Male and Female
Key inclusion criteria	Ability to give patients' consent by written informed consent form Age >= 20 Relapsed or refractory primary/secondary AML diagnosed by 2016 WHO classification Awareness of AML Peripheral blood WT-1 mRNA => 1500 copies/microgram ever before ECOG Performance Status <= 2 Life expectancy >= 12 weeks Submission of preserved specimens at screening and newly collected bone marrow/bone marrow aspiration
Key exclusion criteria	Acute promyelocytic leukemia, BCR-ABL-positive leukemia Central nervous system infiltration/extramedullary AML Sustained non-hematological toxicity ( >= Grade 2) related to prior therapy for AML Clinically relevant graft-versus-host disease required for treatment Duration from prior therapy to administration: 1) Systemic immunosuppressive drugs including steroids <= 14 days 2) Investigational drugs, products, or medical devices <= 4 weeks 3) Hematopoietic stem cell transplantation <= 8 weeks 4) Chemotherapy (excluding hydroxyurea for leukemia control) Laboratory examination <= 14 days prior to registration: 1) AST/ALT >= 3 times upper limit of normal level (ULN) 2) Total serum bilirubin level >= 2 times ULN 3) Lymphocytes (peripheral blood) <= 5.0 x 10^2 /microliter 4) Estimated glomerular filtration rate < 30 mL/min 5) SpO2 < 94% (room air) Other active malignancies Angina pectoris, acute myocardial infarction, congestive heart failure (>= NYHA class 3), or severe abnormality on electrocardiography <= 12 weeks prior to 1st administration Poor control hypertension, interstitial pneumonia, pulmonary fibrosis, chronic obstructive pulmonary disease (>= stage 3) Disseminated intravascular coagulation Active/poor control infection, HIV infection Active hepatitis B/C virus infection, other active liver disease Congenital/acquired immune deficiency Pregnant, possible pregnant, breast feeding women Poor control diabetes mellitus Known hypersensitivity to reagents (human albumin, etc.), additives (galactose/ceramide), antibiotics (streptomycin/gentamicin) and heterologous proteins (fetal bovine serum/porcine trypsin) Principal investigator/co-investigator judgement
Target sample size	18

Research contact person
Name of lead principal investigator	1st name Toyotaka Middle name Last name Kawamata
Organization	The Institute of Medical Science, The University of Tokyo
Division name	Department of Hematology/Oncology, IMSUT Hospital
Zip code	108-8639
Address	4-6-1 Shirokanedai, Minato-ku, Tokyo
TEL	03-5449-8111
Email	dctsm@ims.u-tokyo.ac.jp

Public contact
Name of contact person	1st name Center for Middle name Last name Translational Research
Organization	The Institute of Medical Science, The University of Tokyo
Division name	Center for Translational Research, IMSUT Hospital
Zip code	108-8639
Address	4-6-1 Shirokanedai, Minato-ku, Tokyo
TEL	03-5449-5462
Homepage URL
Email	dctsm@ims.u-tokyo.ac.jp

Sponsor
Institute	IMSUT Hospital, The Institute of Medical Science, The University of Tokyo
Institute
Department

Funding Source
Organization	National Research and Development Institute Agency RIKEN
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	IMSUT hospital Institutional Review Board (IRB), The Institute of Medical Science, The University of Tokyo
Address	4-6-1 Shirokanedai, Minato-ku, Tokyo
Tel	03-5449-5462
Email	imsuttro@ims.u-tokyo.ac.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 07 Month 05 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled	10
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

Progress
Recruitment status	Completed
Date of protocol fixation	2017 Year 03 Month 08 Day
Date of IRB	2020 Year 05 Month 28 Day
Anticipated trial start date	2017 Year 07 Month 05 Day
Last follow-up date	2020 Year 03 Month 03 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 07 Month 05 Day
Last modified on	2020 Year 06 Month 11 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000031964