Myelodysplastic SyndromePhase 2NCT01048034
Evaluation of Azacitidine in Transfusion Dependent Patients With Low-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML)
A Phase 2 study testing Azacitidine and Erythropoetin in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by Nordic MDS Group at 17 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
- What is being tested?
- Azacitidine and Erythropoetin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 17 sitesin 3 countries
- Study dates
- Jan 2010to Aug 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 11Show fewer
- Serum creatinine >2.0 mg/dL (177 micromol/L)
- 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)
- Serum total bilirubin >1.5 mg/dL (26 micromol/L)
- Uncontrolled systemic infection
- Considered not capable of following the study protocol
Trial locations and contacts
17 sites in 3 countries.
17 sites in 3 countries
Sweden10 sites
Department of Medicine, Mälarsjukhuset Hospital
Eskilstuna
Department of medicine, Falun Hospital
Falun
Department of Medicine, Sahlgrenska University Hospital / Östra
Gothenburg
Department of Hematology, Linköping University Hospital
Linköping
Department of Medicine, Sunderbyn Hospital
Luleå
Department of Hematology, Lund University Hospital
Lund
Department of Hematology, Karolinska University Hospital
Stockholm
Department of Medicine, Södersjukhuset Hospital
Stockholm
Department of Medicine, Umeå University Hospital
Umeå
Department of Medicine, Uppsala University Hospital
Uppsala
Denmark5 sites
Department of Hematology, Aalborg University Hospital
Aalborg
Department of Hematology, Aarhus Univsersity Hospital
Aarhus
Department of Hematology, Rigshospitalet Univsersity Hospital
Copenhagen
Department of Hematology, Herlev Hospital
Herlev
Department of Hematology, Odense University Hospital
Odense
Norway2 sites
Department of Medcine, Haukeland University Hospital
Bergen
Department of Hematology, Rikshospitalet University Hospital
Oslo
About this study
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
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.
- Being tested
- Azacitidine, Erythropoetin
- Keywords
- Myelodysplastic syndrome, Chronic myelomonocytic leukemia, Transfusion therapy, Transfusion dependency, Hypomethylating therapy, Azacitidine, DNA-methylation, Epigenetic modifications
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
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
ErythropoetinDrug
For those patients not responding to Azacitidine alone, the combination of Azacitidine and erythropoetin 60 000 U / week for 16 weeks will be given.
| Study group | Role | What they receive |
|---|---|---|
| Azacitidine +/- erythropoetin | Experimental | Not described Receives: Drug: Azacitidine; Drug: Erythropoetin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin level
Measured: Week 28
Number of patients reaching transfusion independency after treatment with Azacitidine
Measured: Week 28
Secondary outcomes (5)
Effect on leucocyte, platelet count
Measured: Week 28 and End of Trial
Effect on bone marrow morphology and cytogenetics
Measured: Week 28 and End of Trial
Number of patients reaching transfusion independency after treatment with Azacitidine and Epo
Measured: End of Trial
Effect on genetic and epigenetic profile
Measured: Week 28
Hemoglobin level
Measured: End of Trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2010 (estimated)
- Study startJan 2010
- Primary completionAug 2012
- Study completionAug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nordic MDS Group
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Magnus Tobiasson, M.D., Study director, Nordic MDS Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01048034
- Sponsor's protocol ID
- NMDSG08A
- Other IDs
- 2009-011483-11, 2009
- Status last verified
- Oct 2013
- First submitted
- 12 Jan 2010
- Last updated
- 29 Oct 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01048034
- Acronym / sponsor ID
- NMDSG08A
- Keywords
- Myelodysplastic syndrome, Chronic myelomonocytic leukemia, Transfusion therapy, Transfusion dependency, Hypomethylating therapy, Azacitidine, DNA-methylation, Epigenetic modifications
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Nordic MDS GroupResearch network
- Study start
- Jan 2010
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 13 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Azacitidine +/- erythropoetin | Experimental | Drug: Azacitidine, Drug: Erythropoetin |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Azacitidine100 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 | Azacitidine +/- erythropoetin |
| ErythropoetinFor those patients not responding to Azacitidine alone, the combination of Azacitidine and erythropoetin 60 000 U / week for 16 weeks will be given. | Drug | Azacitidine +/- erythropoetin |
Primary outcome
Hemoglobin level
Time frame: Week 28
Number of patients reaching transfusion independency after treatment with Azacitidine
Time frame: Week 28
5 secondary outcomes
Effect on leucocyte, platelet count
Time frame: Week 28 and End of Trial
Effect on bone marrow morphology and cytogenetics
Time frame: Week 28 and End of Trial
Number of patients reaching transfusion independency after treatment with Azacitidine and Epo
Time frame: End of Trial
Effect on genetic and epigenetic profile
Time frame: Week 28
Hemoglobin level
Time frame: End of Trial
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Magnus Tobiasson, M.D. study director
Nordic MDS Group
17 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine and Erythropoetin in people with Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia, run by Nordic MDS Group at 17 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Oct 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Sweden, Denmark, Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin level (time frame: Week 28). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2013; AzTrials last refreshed its copy on 7 Nov 2026.