Phase 4
N=100
A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT02145026 ↗Enrolled (actual)
100
Serious AEs
21.2%
Results posted
Apr 2020
Primary outcome: Primary: Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria — 60 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Epoetin beta (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Apr 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria |
60 | — |
| SECONDARY Percentage of Participants With Platelet Response (in Participants With Pre-Treatment Platelets <100*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria |
16 | — |
| SECONDARY Percentage of Participants With Neutrophil Response (in Participants With Pre-Treatment Neutrophil <1.0*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria |
33.3 | — |
| SECONDARY Percentage of Participants With Adverse Events |
48.48 | — |
Summary
This is a Phase IV, prospective, multi-center, open-label study to assess the effectiveness and safety profile of epoetin beta (Recormon®) for treatment of symptomatic anemia in adult participants associated with low/intermediate-1-risk MDS. After screening, eligible participants will be treated with epoetin beta as recommended in the approved label and international guidelines for the use of epoetin in MDS participants and the dosage will be adjusted on the basis of erythroid response.
Eligibility Criteria
Inclusion Criteria
- Adult participants with low or intermediate-1 risk MDS
- No previous treatment with hematopoietic growth factors within 3 months prior to screening
- Symptomatic anemia (hemoglobin <10 g/dL) as determined by investigator
- Serum erythropoietin <500 milliunits/milliliter (mU/mL) within 14 days prior to the first dose of study treatment
- Require no red blood cell transfusion or dependent on <4 units within 8 weeks prior to screening
- Clinically stable for at least 1 month prior to entry into the study
- For female participants of childbearing potential and male participants with partners of childbearing potential, agreement (by participants and/or partner) to use highly effective form(s) of contraception
Exclusion Criteria
- Contraindications and/or known hypersensitivity to the active substance and/or any of the excipients of epoetin beta treatment
- Poorly controlled hypertension as assessed by the investigator
- History of Acute Myeloid Leukemia (AML) or high risk for AML
- Administration of another investigational drug within 1 month before screening or planned during the study period
- Previously documented evidence of Pure Red Cell Aplasia (PRCA)
Data sourced from ClinicalTrials.gov (NCT02145026). 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.