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

Information source: Nordic MDS Group
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Myelodysplastic Syndrome; Chronic Myelomonocytic Leukemia

Intervention: Azacitidine (Drug); Erythropoetin (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: Nordic MDS Group

Official(s) and/or principal investigator(s):
Magnus Tobiasson, M.D., Study Director, Affiliation: Nordic MDS Group

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.

Clinical Details

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 design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Hemoglobin level

Number of patients reaching transfusion independency after treatment with Azacitidine

Secondary outcome:

Effect on leucocyte, platelet count

Effect on bone marrow morphology and cytogenetics

Number of patients reaching transfusion independency after treatment with Azacitidine and Epo

Effect on genetic and epigenetic profile

Hemoglobin level

Eligibility

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

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

Locations and Contacts

Department of Hematology, Aalborg University Hospital, Aalborg, Denmark

Department of Hematology, Aarhus Univsersity Hospital, Aarhus, Denmark

Department of Hematology, Rigshospitalet Univsersity Hospital, Copenhagen, Denmark

Department of Hematology, Herlev Hospital, Herlev, Denmark

Department of Hematology, Odense University Hospital, Odense, Denmark

Department of Medcine, Haukeland University Hospital, Bergen, Norway

Department of Hematology, Rikshospitalet University Hospital, Oslo, Norway

Department of Medicine, Mälarsjukhuset Hospital, Eskilstuna, Sweden

Department of medicine, Falun Hospital, Falun, Sweden

Department of Medicine, Sahlgrenska University Hospital / Östra, Göteborg, Sweden

Department of Hematology, Linköping University Hospital, Linköping, Sweden

Department of Medicine, Sunderbyn Hospital, Luleå, Sweden

Department of Hematology, Lund University Hospital, Lund, Sweden

Department of Hematology, Karolinska University Hospital, Stockholm, Sweden

Department of Medicine, Södersjukhuset Hospital, Stockholm, Sweden

Department of Medicine, Umeå University Hospital, Umeå, Sweden

Department of Medicine, Uppsala University Hospital, Uppsala, Sweden

Additional Information

Starting date: January 2010
Last updated: October 28, 2013

Page last updated: August 23, 2015

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