High-risk MDS
Conditions
Keywords
MDS, Transplant, Azacitidine
Brief summary
Open-label, randomized multicenter phase III non-inferiority study
Detailed description
Open-label, randomized, prospective multicenter phase III study to compare the role of HMT followed by HSCT vs HSCT upfront in HR-MDS with \<10% of BM blasts and of CHT vs HMT followed by HSCT in HR-MDS with \>10% BM blasts in terms of feasibility of HSCT (non-inferiority trial).
Interventions
DRUGAzacitidine
75mg/mq/day subcutaneously for 7 days every 28 days
DRUGStandard Chemotherapy
1. cycle (induction): i.v. 3+7 (Citarabine 200 mg/m2 iv continuous infusion (24 h) for 7 days, Daunorubicine 60 mg/mq iv day 1-3) 2. cycle (consolidation): i.v. 3+7 (Citarabine 200 mg/m2 iv continuous infusion (24 h) for 7 days, Daunorubicine 45 mg/mq iv day 1-3)
PROCEDUREAllogeneic stem cell transplantation
Allogeneic stem cell transplantation
Sponsors
Gruppo Italiano Malattie EMatologiche dell'Adulto
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high 2. Age 18-70 years 3. Previously untreated for HR-MDS 4. HSCT - eligible 5. Life expectancy ≥3 months; 6. Signed written informed consent according to ICH/EU/GCP and national local laws 7. Eastern Cooperative Oncology Group Performance Status Grade of 0-2
Exclusion criteria
1. Acute myeloid leukaemia with \>20% blasts in BM or peripheral blood (PB); 2. concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma); 3. severe renal, cardiac, liver or lung impairment; 4. pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine. 5. HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis; 6. clinically relevant neurological or psychiatric diseases; 7. hypersensitivity (known or suspected) to AZA; 8. prior Treatments: 1. prior investigational drugs (within 30 days); 2. radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months; 3. growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days; 4. androgenic hormones during the previous 14 days; 5. prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS (a previous treatment with Lenalidomide is admitted, provided that lenalidomide had been stopped at least 60 days before enrolment).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of HSCT in terms of proportion of patients who receive HSCT of the total number of randomized patients | 4 years | Split patients in two categories: the feasibility of HSCT (ITT) in patients with HR-MDS with a proportion of bone marrow blasts below 10% and in patients with a proportion of BM blasts equal or greater than 10%. |
Countries
Italy
Contacts
Primary ContactPaola Fazi
Backup ContactEnrico Crea
Outcome results
None listed