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Wilms Tumor Gene (WT1) mRNA-transfected Autologous Dendritic Cell Vaccination for Patients With Acute Myeloid Leukemia (AML): a Phase I Trial

Phase 1
18 Years
Not Enrolling
Acute Myeloid Leukemia (AML)

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Trial Information

Wilms Tumor Gene (WT1) mRNA-transfected Autologous Dendritic Cell Vaccination for Patients With Acute Myeloid Leukemia (AML): a Phase I Trial

Autologous dendritic cell (DC) vaccination is a promising strategy for adjuvant cancer
therapy in the setting of minimal residual disease (MRD). We performed a phase I/II trial in
patients with acute myeloid leukemia (AML) where patients received intradermal injections of
autologous DC loaded with mRNA coding for the Wilms' tumor protein (WT1). WT1 is highly
overexpressed in leukemia and the level of WT1 RNA in peripheral blood is a useful biomarker
for molecular diagnosis en follow-up in the MRD setting. We want to prospectively monitor
WT1 RNA expression in the peripheral blood of vaccinated and non-vaccinated AML patients in
order to evaluate its predictive value as a biomarker for relapse and to assess the clinical
efficacy of DC vaccination in acute myeloid leukemia patients. We believe, on the basis of
already available evidence, that the use of WT1 both as a target for immunotherapy as well
as a biomarker not holds promise to assess the efficacy of new experimental therapeutic
interventions such as DC vaccination.

Inclusion Criteria:

1. Tumor type: Acute Myeloid Leukemia (AML) according to the WHO criteria (ea at least
20% blasts in the marrow). All FAB subtypes except M3. Patients with Myelodysplastic
Syndrome, category of Refractory Anemia with Excess Blasts (RAEB): RAEB I (WHO:
medullary blast count ≤ 10% and a peripheral blast count ≤ 5%) and RAEB II (WHO:
medullary blast count > 10% and/or > 5% peripheral blasts) can be included in the
study in absence of other non-experimental treatment modalities.

2. Extent of disease: remission (partial or complete) or smouldering course. Complete
remission (CR) is defined as no blasts in the peripheral blood and no more than 5%
blasts in the bone marrow. This definition is related to the hematological remission
if it is not specified. Partial remission (PR) is defined as a decrease of at least
50% in the percentage of blasts to 5 to 25% in the bone marrow aspirate. Smouldering
course is defined as a relatively low marrow blast count and slowly progressive

3. Overexpression of WT1 RNA (>50 copies of WT1 per 1000 copies ABL in bone marrow or >2
copy/1000 copies ABL in peripheral blood) as assessed by quantitative RT-PCR at the
time of presentation.

4. Prior treatments : Patients must have received at least one prior antileukemic
chemotherapeutic regimen and must be more than 1 month past the last treatment and/or
6 months past allogeneic/autologous stem cell transplantation.

5. Age: ≥ 18 years

6. High risk of relapse because of (and/or)

- Age > 60 years (if <60 y, no sibling allotransplant donor available)

- Poor risk cytogenetic or molecular markers at presentation

- Hyperleukocytosis at presentation

- Second complete remission after relapse

7. Performance status: WHO PS grade 0-1 (Appendix B)

8. Objectively assessable parameters of life expectancy: more than 3 months

9. Prior and concomitant associated diseases allowed with the exception of underlying
autoimmune disease and positive serology for HIV/HBV/HCV

10. No concomitant use of immunosuppressive drugs

11. Adequate renal and liver function, i.e. creatinin and bilirubin = 1.2 times the upper
limit of normal

12. Absence of any psychological, familial, sociological or geographical condition
potentially hampering compliance with the study protocol and follow-up schedule;
those conditions should be discussed with the patient before registration in the

13. Women of child-bearing potential should use adequate contraception prior to study
entry and for the duration of study participation

Exclusion Criteria:

1. Subjects with concurrent additional malignancy (with exception of Non-melanoma skin
cancers and carcinoma in situ of the cervix)

2. Subjects who are pregnant

3. Subjects who have sensitivity to drugs that provide local anesthesia

4. Age < 18 years

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

acute toxicity of intradermal injections of WT1 mRNA-electroporated autologous dendritic cells

Safety Issue:


Principal Investigator

Zwi Berneman, MD, PHD

Investigator Role:

Study Director

Investigator Affiliation:

University Hospital, Antwerp


Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment

Study ID:

EC 5/6/29



Start Date:

March 2005

Completion Date:

December 2008

Related Keywords:

  • Acute Myeloid Leukemia (AML)
  • AML in remission
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid