COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

Evaluation of Azacitidine in Transfusion Dependent Patients With Low-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01048034
Recruitment Status : Completed
First Posted : January 13, 2010
Last Update Posted : October 29, 2013
Information provided by (Responsible Party):
Nordic MDS Group

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Chronic Myelomonocytic Leukemia Drug: Azacitidine Drug: Erythropoetin Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : January 2010
Actual Primary Completion Date : August 2012
Actual Study Completion Date : August 2012

Arm Intervention/treatment
Experimental: Azacitidine +/- erythropoetin Drug: Azacitidine
100 mg / m(2) subcutaneously day 1-5 every 4 weeks for 6 cycles. Another three cycles will be given together with epo for those not responding to the first 6 cycles of Azacitidine

Drug: Erythropoetin
For those patients not responding to Azacitidine alone, the combination of Azacitidine and erythropoetin 60 000 U / week for 16 weeks will be given.

Primary Outcome Measures :
  1. Hemoglobin level [ Time Frame: Week 28 ]
  2. Number of patients reaching transfusion independency after treatment with Azacitidine [ Time Frame: Week 28 ]

Secondary Outcome Measures :
  1. Effect on leucocyte, platelet count [ Time Frame: Week 28 and End of Trial ]
  2. Effect on bone marrow morphology and cytogenetics [ Time Frame: Week 28 and End of Trial ]
  3. Number of patients reaching transfusion independency after treatment with Azacitidine and Epo [ Time Frame: End of Trial ]
  4. Effect on genetic and epigenetic profile [ Time Frame: Week 28 ]
  5. Hemoglobin level [ Time Frame: End of Trial ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01048034

Show Show 17 study locations
Sponsors and Collaborators
Nordic MDS Group
Layout table for investigator information
Study Director: Magnus Tobiasson, M.D. Nordic MDS Group
Layout table for additonal information
Responsible Party: Nordic MDS Group Identifier: NCT01048034    
Other Study ID Numbers: NMDSG08A
2009-011483-11 ( EudraCT Number )
First Posted: January 13, 2010    Key Record Dates
Last Update Posted: October 29, 2013
Last Verified: October 2013
Keywords provided by Nordic MDS Group:
Myelodysplastic syndrome
Chronic myelomonocytic leukemia
Transfusion therapy
Transfusion dependency
Hypomethylating therapy
Epigenetic modifications
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Leukemia, Myelomonocytic, Juvenile
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Leukemia, Myeloid
Myelodysplastic-Myeloproliferative Diseases
Epoetin Alfa
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors