Patients with high risk myelodysplastic syndrome or acute myeloid leukemia MedDRA version: 21.0 Level: LLT Classification code 10000886 Term: Acute myeloid leukemia System Organ Class: 100000004864 MedDRA version: 21.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864
Conditions
Interventions
Product Code: ASTX727
Pharmaceutical Form: Tablet
INN or Proposed INN: CEDAZURIDINE
CAS Number: 1141397-80-9
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
IN
Sponsors
Groupe Francophone des Myélodysplasies (GFM)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Patients aged from 18 to 70 years - MDS or AML with unfavorable genetics defined as follow: -4 or more cytogenetic abnormalities or -3 cytogenetic abnormalities and TP53 or other unfavorable mutations (ASXL1, RUNX1) or -3 cytogenetic abnormalities and monosomal karyotype or -mutations involving EVI1 - AML patients should have received chemotherapy - Marrow blast =65 years) yes F.1.3.1 Number of subjects for this age range 40
Exclusion criteria
Exclusion criteria: - ECOG 3 or more - Cancer less than 2 years before inclusion or cancer not in remission the last 2 years before inclusion (except in situ cancer or baso cellular cancer) - Cardiac failure with EF 150 µmol/L - Liver enzyme > 3 N - Conjugated bilirubinemia > 25 µmol/L - MDS occurring in patients with Fanconi anemia or congenital dyskeratosis - Proliferative disease in patients not in remission: WBC > 15 G/L or use of continuous cytotoxic to maintain WBC < 15 G/L - AML with marrow or peripheral blast count higher than 10% after chemotherapy - No contraception - Pregnant or breastfeeding women
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Achieving a disease-free survival of 35% (expected 12%), 12 months after transplant;Secondary Objective: -Proportion of patients receiving allo SCT -Proportion of patients eligible for post-transplant oral decitabine -Proportion of patients eligible for post-transplant DLI -Grade III/IV toxicities -Non-relapse mortality -Proportion of patients completing treatment schedule -Overall survival at 12 and 24 months, DFS at 24 months after transplant ;Primary end point(s): DFS at 1 year post transplant;Timepoint(s) of evaluation of this end point: At 1 year | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Overall survival from the date of transplantation and from the date of inclusion at 1 year and 2 years - Risk factors for DFS, OS NRM at 1 year and 2 years ;Timepoint(s) of evaluation of this end point: At 1 and 2 years | — |
Countries
France
Contacts
Public ContactCRA coordinator
GFM
Outcome results
None listed