A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.

Study Overview
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Brief Summary
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This single arm study will evaluate the efficacy and safety of a combination of NeoRecormon, CellCept and prednisone in patients with low or moderate risk myelodysplastic syndromes (MDS). In the first phase of the study, patients will receive CellCept (1g p.o. twice daily) plus prednisone. After 3 months, if patients have not responded to treatment, NeoRecormon (30000 IU/week, s.c.) will be added to the treatment regimen. If there is no response to NeoRecormon after 6 weeks, the dose will be increased to 60000 IU/week. The anticipated time on study treatment is 3-12 months, and the target sample size is <100 individuals.

Official Title
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An Open Label Study of the Effects of a Combination of NeoRecormon, CellCept and Prednisone on Hematological Parameters and Cytogenesis in Patients With Low or Intermediate Risk Myelodysplastic Syndromes.¿

Conditions
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Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: Mycophenolate mofetil
* Drug: Prednisone
* Drug: Erythropoietin Beta


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: adult patients, >=18 years of age; diagnosis of MDS, according to International Prognostic Scoring System (IPSS) criteria; low or intermediate risk, who are not candidates for treatment with growth factors, or who have not responded to these treatments. Exclusion Criteria: previous treatment with CellCept, or any erythropoietin-stimulating drug; diagnosis of proliferative chronic myelomonocytic leukemia; prior or concomitant malignancies other than MDS, with the exception of basocellular, spinocellular or adequately treated in situ cervical cancer, in the past 3 years; biological antitumor and myelosuppressive treatment within 28 days before start of study; bone marrow precursor cell transplantation previous to study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mycophenolate Mofetil + Prednisone + Erythropoietin Beta<br>Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12. | Drug: Mycophenolate mofetil<br>* 1 gm twice daily orally until end of study.<br>* Other names: MMF;Drug: Prednisone<br>* 10 mg/day orally until end of study.<br>Drug: Erythropoietin Beta<br>* Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.<br>* Other names: NeoRecormon;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement | International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) <11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment. | Up to approximately 2 years | 
| Mean Number of Blood Transfusions Per Visit |  | Up to approximately 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With at Least One Adverse Event (AE) | An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. | Up to approximately 2 years |