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

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.¿

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 Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Mycophenolate mofetil; Drug: Prednisone; Drug: Erythropoietin Beta

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• Spain
  • Barakaldo, Spain, 48903
  • Barcelona, Spain, 08036
  • Barcelona, Spain, 08003
  • Barcelona, Spain, 08035
  • Barcelona, Spain, 08025
  • Cádiz, Spain, 11009
  • Madrid, Spain, 28040
  • Palma de Mallorca, Spain, 07198

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mycophenolate Mofetil + Prednisone + Erythropoietin Beta; 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; 1 gm twice daily orally until end of study.; Other Names: CellCept; MMF; Drug: Prednisone; 10 mg/day orally until end of study.; Drug: Erythropoietin Beta; Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.; Other Names: NeoRecormon

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start: August 1, 2007
• Primary Completion (Actual): June 1, 2009
• Study Completion (Actual): June 1, 2009

Study Registration Dates

• First Submitted: October 29, 2007
• First Submitted That Met QC Criteria: October 29, 2007
• First Posted (Estimate): October 30, 2007

Study Record Updates

• Last Update Posted (Estimate): July 25, 2016
• Last Update Submitted That Met QC Criteria: June 24, 2016
• Last Verified: June 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Anti-Inflammatory Agents; Antineoplastic Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antitubercular Agents; Hematinics; Antibiotics, Antitubercular; Epoetin Alfa; Prednisone; Mycophenolic Acid
• Other Study ID Numbers: ML20559

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No