Clinical trial · Interventional
Evaluation of Azacitidine in Transfusion Dependent Patients With Low-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML)
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Azacitidine has proved prolonged overall survival in patients with high-risk MDS. Minor pilot studies have shown that treatment with Azacitidine can induce transfusion independency in previous transfusion dependent patients with low-risk MDS. This study will evaluate the effect of Azacitidine in transfusion dependent patients with low-risk MDS (IPSS low or int-1) or low risk CMML. Included patients should first have failed, or considered not being eligible to, treatment with EPO +/- G-CSF. Our hypothesis is that Azacitidine can lead to transfusion independency in this group of patients. Those patients who do not respond to treatment with Azacitidine alone, will be given treatment with the combination of Azacitidine and EPO where our hypothesis is that Azacitidine can restore sensitivity to EPO.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Chronic Myelomonocytic Leukemia | Chronic Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Azacitidine | Drug | Azacitidine | ALIAS |
| Erythropoetin | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Azacitidine +/- erythropoetin
- interventionNames
- Drug: Azacitidine
- Drug: Erythropoetin
Primary outcomes (2)
- measure
- Hemoglobin level
- timeFrame
- Week 28
- measure
- Number of patients reaching transfusion independency after treatment with Azacitidine
- timeFrame
- Week 28
Secondary outcomes (5)
- measure
- Effect on leucocyte, platelet count
- timeFrame
- Week 28 and End of Trial
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * 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 model (see Hellstrom-Lindberg et al, Br J Haematol 99:344-51 1997)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. Exclusion Criteria: * 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
References
Publications (0)
Data not yet available