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

Hoffmann-La Roche10 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Syndromes

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (8)

Unnamed facility

Barakaldo, Spain

Unnamed facility

Barcelona, Spain

Unnamed facility

Barcelona, Spain

Unnamed facility

Barcelona, Spain

Unnamed facility

Barcelona, Spain

Unnamed facility

Cadiz, Spain

Unnamed facility

Madrid, Spain

Unnamed facility

Palma de Mallorca, Spain

Outcomes

Primary Outcomes

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.

Time frame: Up to approximately 2 years

Mean Number of Blood Transfusions Per Visit

Time frame: Up to approximately 2 years

Secondary Outcomes

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.

Time frame: Up to approximately 2 years