A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)

Study Overview
=================
Brief 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.

Official Title
-----------------
A Prospective Open-Label Study of the Effectiveness of Epoetin Beta for Treating Anemic Patients With Low/Intermediate-1-Risk Myelodysplastic Syndrome (MDS)

Conditions
-----------------
Myelodysplastic Syndromes

Intervention / Treatment
-----------------
* Drug: Epoetin beta


Participation Criteria
=================
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)

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Epoetin Beta<br>Participants will receive epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Response will be firstly evaluated at Week 4 and the subsequent dose will be based on the response: if hemoglobin level reaches greater than or equal to (>/=)12 grams per deciliter (g/dL) at any time, epoetin beta will be discontinued until hemoglobin levels are less than or equal to (</=) 10 g/dL; if the hemoglobin level increases less than (<) 1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 60,000 IU per week epoetin beta will be administered SC until Week 12; if the hemoglobin level increases >/=1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 30,000 IU per week epoetin beta will be continued until Week 12. | Drug: Epoetin beta<br>* Epoetin beta 30,000 or 60,000 IU per week SC injection<br>* Other names: Recormon;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria | Erythroid response at Week 12 according to IWG 2006 criteria was defined as a hemoglobin (Hb) increase of >/= 1.5 grams/deciliter (g/dL), and a reduction of units of red blood cell (RBC) transfusions by at least 4 transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hb of </= 9.0 g/dL pre-treatment were counted in the RBC transfusion response evaluation. | Week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Platelet response according to IWG 2006 criteria was defined as an absolute increase of >/= 30x10^9/L for participants starting with >20x10^9/L platelets. | Week 12 | 
| 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 | Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of >0.5x10^9/L. | Week 12 | 
| Percentage of Participants With Adverse Events |  | From signing of informed consent up to 4 weeks after last dose (up to 18 weeks) |