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

Roche

Status and phase: Completed; Phase 4

Conditions: Myelodysplastic Syndromes

Treatments: Drug: Epoetin beta

Study type: Interventional

Funder types: Industry

Identifiers

NCT02145026

ML29005

Details and patient eligibility

About

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.

Enrollment: 100 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Epoetin Beta

Experimental group

Description:

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.

Treatment:

Drug: Epoetin beta