A Phase I/II Study to Assess the Safety and Immunogenicity of WT1-A10 + AS01B Antigen-Specific Cancer Immunotherapeutic (ASCI) Combined With Infusions of ex Vivo Regulatory T Cells Depleted T Lymphocytes in in Vivo Regulatory T Cells Depleted Patients as Post-consolidation Therapy for Adult Patients With WT1-positive Acute Myeloid Leukemia (AML) in CR1 (for High Risk Patients) or in CR2 or CR3 Who Are Not Eligible for Allogeneic Stem Cell Transplantation (SCT).
1. The patient has cytologically proven AML, as defined by the WHO classification. The
leukemia is a de novo or a secondary leukemia.
2. The patient is in complete morphologic remission Note: Cytogenetic CR (CRc) or
molecular CR (CRm) is not required.
- AML patients in first complete remission (CR1) who are not eligible for
allo-HSCT following the institution's standard of care(except the favourable
genetic group subset which is excluded from this study).
- All AML patients in second or third complete morphological remission(CR2 or CR3)
who are not eligible for allo-HSCT.
3. The patient received the following therapy according to the institution's standard of
- For patients ≤ 60 years old, at least two cycles of intensive chemotherapy
(induction and consolidation)
- For patients > 60 years old, at least one induction chemotherapy. Any patients
with severe co-morbidity for which consolidation is unacceptable, can receive
only one induction therapy.
4. The patient's blasts cells show over-expression of WT1 transcripts, detected in
peripheral blood by qRT-PCR at diagnosis or at first relapse.
5. Written informed consent has been obtained prior to the performance of any
6. The patient is ≥ 18 years of age at the time of signing of the ICF.
7. ECOG performance status of 0, 1, or 2 at the time of enrollment.
8. Adequate hepatic and renal function defined as:
- Serum bilirubin < 1.5 times the Upper Limit of Normal (ULN).
- Serum alanine aminotransferase ALAT < 2.5 times the ULN.
- Calculated creatinine clearance > 40 mL/min.
9. If the patient is female, then she must be of non-childbearing potential, i.e., have
a current tubal ligation, hysterectomy, ovariectomy or be post-menopausal, or if she
is of childbearing potential, then she must practice adequate contraception for 30
days prior to treatment administration, have a negative pregnancy test, and continue
such precautions for two months after completion of the treatment administration
10. Under the investigator criteria, the patient is able to comply with the protocol
requirements during the duration of the study.
11. In the investigator's opinion and in compliance with the Institution hematology
guidance, the patient should not be eligible for an approved standard of care such as
induction with chemotherapy or allo-HSCT.
1. The patient is in morphologic leukemia-free state or in morphologic complete
remission but with incomplete blood count recovery as defined by IWG Response
2. The patient is in CR1 and is in the category of low-risk for relapse patients, i.e.
belong to the favourable genetic group subset .
3. The patient was diagnosed with leukemic central nervous system (CNS) disease (E.g.
before chemotherapy) or presents neurological symptoms at baseline suggestive of a
4. The patient has received, is receiving (or is due to receive) allo-HSCT.
5. The patient has (or has had) concomitant malignancies, except effectively treated
malignancy that is considered by the investigator highly likely to have been cured.
6. The patient is known to be human immunodeficiency virus (HIV)-positive.
7. The patient has symptomatic autoimmune disease such as, but not limited to, multiple
sclerosis, lupus, rheumatoid arthritis and inflammatory bowel disease.
8. The patient has a history of allergic reactions likely to be exacerbated by any
component of the study investigational product.
9. The patient has other concurrent severe medical problems, unrelated to the
malignancy, that would significantly limit full compliance with the study or expose
the patient to unacceptable risk.
10. The patient has congestive heart failure, symptomatic coronary artery disease, or
previous myocardial infarction.
11. The patient has psychiatric or addictive disorders that may compromise his/her
ability to give informed consent, or to comply with the study procedures.
12. The patient has received any investigational or non-registered medicinal product
other than the study medication within 30 days preceding the first dose of study
medication, or plans to receive such a drug during the study period.
13. The patient requires concomitant treatment with systemic corticosteroids or any other
immunosuppressive agents. The use of prednisone, or equivalent, < 0.5 mg/kg/day
(absolute maximum 40 mg/day), inhaled corticosteroids or topical steroids is
14. The patient has an active infection and/or is receiving antibiotics. The patient has
received i.v. administration of antibiotics within two weeks prior to first study
treatment or oral antibiotics within one week prior to first study treatment.
15. For female patients: the patient is pregnant or lactating.