Phase 2
N=10
A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.
Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00551291 ↗Enrolled (actual)
10
Serious AEs
40.0%
Results posted
Jun 2016
Primary outcome: Primary: Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement — 50.00; 71.43; 100.00 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Mycophenolate mofetil (Drug); Prednisone (Drug); Erythropoietin Beta (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Jun 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement |
50.00; 71.43; 100.00 | — |
| PRIMARY Mean Number of Blood Transfusions Per Visit |
4.13; 5.83; 2.80; 2.33 | — |
| SECONDARY Percentage of Participants With at Least One Adverse Event (AE) |
90.00 | — |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT00551291). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.