A phase IIa study of aAVC-WT1 therapy to treat acute myeloid leukemia with MRD-positive complete remission or partial remission and high-risk myelodysplastic syndromes

JRCT ID: jRCT2033200305

Registered date:15/01/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: AML with MRD-positive complete remission or partial remission and high-risk MDS
• Date of first enrollment: 08/11/2021
• Target sample size: 18
• Study type: Interventional
• Intervention(s): Intravenous administraion of aAVC-WT1

Outcome(s)

Primary Outcome

Safety
1)Exploration of adverse events
2)Statistical analysis on examinations related to safety evaluation

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Ability to give patients' consent by written informed consent form
2.Age >= 20
3.AML with MRD-positive complete remission or partial remission and high-risk MDS by 2016 WHO classification
4.Awareness of AML or MDS
5.Peripheral blood WT-1 mRNA => 50 copies/microgram ever before
6.In patients with Hematological CR, one of the following 1) and 2) was observed :
1) Blood WT-1 mRNA => 50 copies/microgram
2) In the quantitative analysis of disease-specific mRNAs (one or more of RUNX1-RUNX1T1, DEK-NUP214, NUP98-HOXA9, CBFB-MYH11, KMT2A-MLLT3, KMT2A-AFDN) caused by chromosomal abnormalities using bone marrow or peripheral blood, disease-specific mRNAs => 50 copies/microgram.
7.ECOG Performance Status <= 2
8.Life expectancy >= 12 weeks

Exclude criteria

1.Acute promyelocytic leukemia, BCR-ABL-positive leukemia
2.Central nervous system infiltration/extramedullary AML
3.Sustained non-hematological toxicity ( >= Grade 2) related to prior therapy for AML
4.Clinically relevant graft-versus-host disease required for treatment
5.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)
5) SARS-CoV-2 Vaccination within 2 weeks (Preferably for one month.)
(Do not administer aAVC-WT1 while the adverse event(s) persist, even after a patient cleared this criterion.)
6.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)
7.Other active malignancies
8.Angina pectoris, acute myocardial infarction, congestive heart failure (>= NYHA class 3), or severe abnormality on electrocardiography <= 12 weeks prior to 1st administration
9.Poor control hypertension, interstitial pneumonia, pulmonary fibrosis, chronic obstructive pulmonary disease (>= stage 3)
10.Disseminated intravascular coagulation
11.Active/poor control infection, HIV infection
12.Active hepatitis B/C virus infection, other active liver disease
13.Congenital/acquired immune deficiency
14.Pregnant, possible pregnant, breast feeding women
15.Poor control diabetes mellitus
16.Known hypersensitivity to reagents (human albumin, etc.), additives (galactose/ceramide), antibiotics (streptomycin/gentamicin) and heterologous proteins (fetal bovine serum/porcine trypsin)
17.Principal investigator/co-investigator judgement

Related Information

• Primary Sponsor: Fujii Shinichiro