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

Roche

Status and phase: Completed; Phase 2

Conditions: Myelodysplastic Syndromes

Treatments: Drug: Prednisone; Drug: Erythropoietin Beta; Drug: Mycophenolate mofetil

Study type: Interventional

Funder types: Industry

Identifiers

NCT00551291

ML20559

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Mycophenolate Mofetil + Prednisone + Erythropoietin Beta

Experimental group

Description:

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.

Treatment:

Drug: Prednisone

Drug: Erythropoietin Beta

Drug: Mycophenolate mofetil