Back to resultsA Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
Primary Purpose
Myelodysplastic Syndromes
Status
Completed
Phase
Phase 4
Locations
Thailand
Study Type
Interventional
Intervention
Epoetin beta
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndromes
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)
Sites / Locations
- King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine
- Rajavithi Hospital; Medicine
- Ramathibodi Hospital; Division of Hematology, Department of Medicine
- Siriraj Hospital; Division of Hematology, Department of Medicine
- Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine
- Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine
- Khonkean Regional Hospital; Medicine
- Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology
- Naresaun University hospital
- Sapprasitthiprasong Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Epoetin Beta
Arm 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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
Percentage of Participants With Adverse Events
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02145026
Brief Title
A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
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)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2020
Overall Recruitment Status
Completed
Study Start Date
August 6, 2014 (Actual)
Primary Completion Date
April 9, 2019 (Actual)
Study Completion Date
April 9, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Epoetin Beta
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Epoetin beta
Other Intervention Name(s)
Recormon
Intervention Description
Epoetin beta 30,000 or 60,000 IU per week SC injection
Primary Outcome Measure Information:
Title
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
Description
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.
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
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
Description
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.
Time Frame
Week 12
Title
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
Description
Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of >0.5x10^9/L.
Time Frame
Week 12
Title
Percentage of Participants With Adverse Events
Time Frame
From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Rajavithi Hospital; Medicine
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Ramathibodi Hospital; Division of Hematology, Department of Medicine
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Siriraj Hospital; Division of Hematology, Department of Medicine
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Facility Name
Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine
City
Khon Kaen
ZIP/Postal Code
40002
Country
Thailand
Facility Name
Khonkean Regional Hospital; Medicine
City
Khon Kean
ZIP/Postal Code
40000
Country
Thailand
Facility Name
Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology
City
Pathumthani
ZIP/Postal Code
12120
Country
Thailand
Facility Name
Naresaun University hospital
City
Phitsanulok
ZIP/Postal Code
65000
Country
Thailand
Facility Name
Sapprasitthiprasong Hospital
City
Ubon Ratchathani
ZIP/Postal Code
34000
Country
Thailand
12. IPD Sharing Statement
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A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
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