Clinical and biological evaluation of azacitidine in transfusion-dependent patients with Low and intermediate-1 risk MDS, and low-risk CMML, who are either refractory to or not eligible for treatment with erythropoietin +/- G-CSF

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

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 < 10% marrow blasts or RARS-T
• Patients with high or intermediate probability for response according to the predictive model2 should be refractory to EPO / darbepoetin (equivalent to > 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

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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