AML: Acute Myeloid Leukemia/ cancer of blood and bone marrow
Conditions
Interventions
CBDC-vaccination (day 0, day 14 and day 28):
Patients will receive three WT1 15-mer-peptide pool loaded CBDC-vaccinations
starting at 8 weeks post-CBT every 2 weeks (hence week 8, 10 and 12). The CBD
Sponsors
Prinses Máxima Centrum voor Kinderoncologie
Eligibility
Age
No minimum to 64 Years
Inclusion criteria
Inclusion criteria: - AML patients eligible for allo-HCT according to standard-of-care guidelines, with overexpression of WT1 mRNA in an AML sample (>50 copies WT1/10^4 copies ABL for PB, and >250 copies WT1/10^4 copies ABL for BM) taken at diagnosis and/or relapse (re-)induction chemotherapy. - indication for CB-HCT according to the Prinses Maxima Centrum / UMC Utrecht guidelines - CB selection criteria: the 80% fraction of the unit should contain a minimum total nucleatd cell number of 3x10^7 NC/Kg criteria for any match grade (before cryo-preservation). Preferable CD34+/Kg dose: >1x10e5 in the 80% fraction - The whole CB unit should contain more than 7.5x10^6 total CD34+ before freeze - Karnofsky/Lansky score >=70 - Age limits for part A (safety run) only: >=12 - 16 years of age), part B 0-
Exclusion criteria
Exclusion criteria: -Patients who are preganant or breast-feeding or unwilling to use adequate contraceptive methods -Known allergies to compound used in the CBDC production process or the local anesthetic lidocaïne-tetracain (Rapydan®) and EMLA® (lidocaine/ prilocaine) plasters -Patients included in other intervention studies influencing the endpoints of this study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints: Part A: Safety: Occurrence of DLTs from the first vaccination (t=0) until 84 days after the third CBDC vaccination Part B: Activity: One-year WT1+ AML relapse-free survival rate from the time of the first vaccination as compared to historical controls. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints (part A): - Treatment emergent adverse events (TEAEs), those with initial onset or increasing in severity after the first vaccination. - One-year cumulative incidence of WT1-specific immunity after the first vaccination. - One-year overall survival rate, from the time of first vaccination - One-year WT1+ AML relapse-free survival rate, from the time of first vaccination. Secondary endpoints (part B): TEAEs, those with initial onset or increasing in severity after the first vaccination. - One-year cumulative incidence of WT1-specific immunity after the first vaccination. - One-year overall survival rate from the time of first vaccination. Exploratory endpoints (part B): - Changes in general immune parameters between those samples taken before and those taken after the first vaccination until one year of follow-up. - Expression of inhibitory (immune checkpoint) molecules on the AML in the case of relapse occurring after the first vaccination until one year of follow-up - WT1 T cell responses in the bone marrow - Early cost effectiveness analysis | — |
Countries
Netherlands
Outcome results
None listed