Low and intermediate-1 risk MDS, and low-risk CMML. MedDRA version: 12.0 Level: LLT Classification code 10009018 Term: Chronic myelomonocytic leukaemia MedDRA version: 12.0 Level: LLT Classification code 10028533 Term: Myelodysplastic syndrome
Conditions
Sponsors
NMDSG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects must satisfy the following criteria to be enrolled in the study: • Must be >=18 years of age at the time of signing the informed consent form • MDS at IPSS Low or Int-1, or mixed MDS/MPD; either CMML with 60 000 U of EPO / week for > 8 weeks) followed by EPO + G-CSF for > 8 weeks, or biosimilar drugs in equipotent doses, or EPO + G-CSF upfront for 8 weeks. Patients with low probability for response according to the predictive model, could be included without prior EPO/G-CSF treatment • Transfusion need >4 units over the last 8 weeks, or >8 units over the last 26 weeks. • Subject has signed the informed consent document. • Men and women of childbearing potential must use effective contraception during, and for up to 3 months after treatment. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Potential subjects who meet any of the following criteria will be excluded from participating in the study: • Pregnant or lactating females. • Patients who are eligible for curative treatment • Expected survival less than 24 weeks. • Symptomatic thrombocytopenia / active bleeding • Patients with JAK-2 positive RARS-T if eligible for new investigational drugs • Serum biochemical values as follows 1. Serum creatinine >2.0 mg/dL (177 micromol/L) 2. Serum aminotransferase (AST)/serum glutamic-oxaloacetic transaminase (SGOT) or alanine transaminase (ALT)/serum glutamate pyruvate transaminase (SGPT) >3.0 x upper limit of normal (ULN) 3. Serum total bilirubin >1.5 mg/dL (26 micromol/L) • Uncontrolled systemic infection • Considered not capable of following the study protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To assess the clinical efficacy, in terms of transfusion independence, of 6 cycles (24 weeks) of azacitidine given in transfusion-dependent patients with MDS and IPSS score low, intermediate-1 or low-risk CMML who are either refractory to or not eligible for treatment with erythropoietin +/- G-CSF (predictive group “poor” according to the predictive model) •To assess effect of treatment on haemoglobin level;Secondary Objective: To assess the following; •Effect of treatment on white blood cells (WBC) and platelet (Plt) counts •Effect of treatment on marrow morphology and cytogenetics •Effect of treatment on genetic and epigenetic profiling of marrow cells •Effect of treatment with erythropoietin in patients not receiving TI at week 28 •Follow-up regarding survival and AML transformation until death ;Primary end point(s): •Effect of treatment on haemoglobin level at week 28 •Number of patients reaching transfusion-independency, TI, at week 28, defined as no transfusion needed during week 20-27 | — |
Countries
Denmark, Sweden
Outcome results
None listed