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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Epoetin Beta | Experimental | 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 Ë‚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 Ë‚12 g/dL, a 30,000 IU per week epoetin beta will be continued until Week 12. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Epoetin beta | Drug | Epoetin beta 30,000 or 60,000 IU per week SC injection |
|
| 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 \ | Week 12 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine | Bangkok | 10330 | Thailand | |||
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| ID | Title | Description |
|---|---|---|
| FG000 | Epoetin Beta | Participants received epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Initial response was evaluated at Week 4 and the subsequent dose was based on response: for hemoglobin levels >/= 12 grams per deciliter (g/dL), epoetin beta was discontinued until levels were </= 10 g/dL; for hemoglobin levels <12 g/dL and that increased less than 1 g/dL, 60,000 IU of epoetin beta were administered until Week 12; and for hemoglobin levels <12 g/dL and that increased >/= 1 g/dL, 30,000 IU of epoetin beta were administered until Week 12. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
1 participant withdrew at the baseline visit prior to study treatment and was not included in baseline, safety, and intent-to-treat (ITT) analyses.
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| ID | Title | Description |
|---|---|---|
| BG000 | Epoetin Beta | Participants received epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Initial response was evaluated at Week 4 and the subsequent dose was based on response: for hemoglobin levels >/= 12 grams per deciliter (g/dL), epoetin beta was discontinued until levels were </= 10 g/dL; for hemoglobin levels <12 g/dL and that increased less than 1 g/dL, 60,000 IU of epoetin beta were administered until Week 12; and for hemoglobin levels <12 g/dL and that increased >/= 1 g/dL, 30,000 IU of epoetin beta were administered until Week 12. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 \ | Efficacy analysis was performed on the intent-to-treat (ITT) population, defined as all enrolled participants who received at least one dose of study medication. | Posted | Count of Participants | Participants | Week 12 |
|
Approximately 56 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Epoetin Beta | Participants received epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Initial response was evaluated at Week 4 and the subsequent dose was based on response: for hemoglobin levels >/= 12 grams per deciliter (g/dL), epoetin beta was discontinued until levels were </= 10 g/dL; for hemoglobin levels <12 g/dL and that increased less than 1 g/dL, 60,000 IU of epoetin beta were administered until Week 12; and for hemoglobin levels <12 g/dL and that increased >/= 1 g/dL, 30,000 IU of epoetin beta were administered until Week 12. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
Data for the secondary efficacy endpoints (% of participants with platelet response and % of participants with neutrophil response) is not conclusive given the small number of participants meeting the evaluation criteria for each endpoint.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Hoffmann-La Roche | 1-800-821-8590 | genentech@druginfo.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 12, 2016 | Mar 19, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 21, 2019 | Mar 19, 2020 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C103998 | epoetin beta |
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| 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) |
| Rajavithi Hospital; Medicine |
| Bangkok |
| 10400 |
| Thailand |
| Ramathibodi Hospital; Division of Hematology, Department of Medicine | Bangkok | 10400 | Thailand |
| Siriraj Hospital; Division of Hematology, Department of Medicine | Bangkok | 10700 | Thailand |
| Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine | Chiang Mai | 50200 | Thailand |
| Khonkean Regional Hospital; Medicine | Khon Kaen | 40000 | Thailand |
| Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine | Khon Kaen | 40002 | Thailand |
| Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology | Pathum Thani | 12120 | Thailand |
| Naresaun University hospital | Phitsanulok | 65000 | Thailand |
| Sapprasitthiprasong Hospital | Ubon Ratchathani | 34000 | Thailand |
| Physician Decision |
|
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| 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 | 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. | Efficacy analysis was performed on the intent-to-treat (ITT) population, defined as all enrolled participants who received at least one dose of study medication. | Posted | Number | Percentage of Participants | Week 12 |
|
|
|
| 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 | Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of >0.5x10^9/L. | Efficacy analysis was performed on the intent-to-treat (ITT) population, defined as all enrolled participants who received at least one dose of study medication. | Posted | Number | Percentage of Participants | Week 12 |
|
|
|
| Secondary | Percentage of Participants With Adverse Events | The safety population included all participants that received at least one dose of study medication. | Posted | Number | Percentage of Participants | From signing of informed consent up to 4 weeks after last dose (up to 18 weeks) |
|
|
|
| 4 |
| 99 |
| 21 |
| 99 |
| 48 |
| 99 |
| Congestive heart failure | Cardiac disorders | Non-systematic Assessment |
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| Cardiac tamponade | Cardiac disorders | Non-systematic Assessment |
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| Cardiogenic shock | Cardiac disorders | Non-systematic Assessment |
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| Tachyarrhythmia | Cardiac disorders | Non-systematic Assessment |
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| Gastrointestinal bleeding | Gastrointestinal disorders | Non-systematic Assessment |
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| Esophageal varices | Gastrointestinal disorders | Non-systematic Assessment |
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| Abscess oral | Infections and infestations | Non-systematic Assessment |
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| Hospital acquired pneumonia | Infections and infestations | Non-systematic Assessment |
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| Lobar pneumonia | Infections and infestations | Non-systematic Assessment |
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| Necrotizing fasciitis | Infections and infestations | Non-systematic Assessment |
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| Pneumonia | Infections and infestations | Non-systematic Assessment |
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| Sepsis | Infections and infestations | Non-systematic Assessment |
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| Septic shock | Infections and infestations | Non-systematic Assessment |
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| Syphilis | Infections and infestations | Non-systematic Assessment |
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| Recurrent urinary tract infection | Infections and infestations | Non-systematic Assessment |
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| Urinary tract infection | Infections and infestations | Non-systematic Assessment |
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| Urosepsis | Infections and infestations | Non-systematic Assessment |
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| Hyperglycemia | Metabolism and nutrition disorders | Non-systematic Assessment |
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| Hypokalemia | Metabolism and nutrition disorders | Non-systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | Non-systematic Assessment |
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| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
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| Hypertension worsened | Vascular disorders | Non-systematic Assessment |
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| Hypertensive emergency | Vascular disorders | Non-systematic Assessment |
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| Aphthous ulcer | Gastrointestinal disorders | Non-systematic Assessment |
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| Constipation | Gastrointestinal disorders | Non-systematic Assessment |
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| Hemorrhoids | Gastrointestinal disorders | Non-systematic Assessment |
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| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
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| Short-bowel syndrome | Gastrointestinal disorders | Non-systematic Assessment |
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| Anemia | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Eosinophilia | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Anemia aggravated | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Anxiety | General disorders | Non-systematic Assessment |
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| Leg pain | General disorders | Non-systematic Assessment |
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| Lymphatic obstruction | General disorders | Non-systematic Assessment |
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| Closed fracture | General disorders | Non-systematic Assessment |
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| Elevated blood pressure | General disorders | Non-systematic Assessment |
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| Fatigue | General disorders | Non-systematic Assessment |
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| Gallstones | General disorders | Non-systematic Assessment |
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| Headache | General disorders | Non-systematic Assessment |
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| Increased blood pressure | General disorders | Non-systematic Assessment |
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| Uric acid level increased | General disorders | Non-systematic Assessment |
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| Insomnia | General disorders | Non-systematic Assessment |
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| Leg edema | General disorders | Non-systematic Assessment |
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| Low back pain | General disorders | Non-systematic Assessment |
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| Epistaxis | General disorders | Non-systematic Assessment |
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| Rash | General disorders | Non-systematic Assessment |
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| Purpura senile | General disorders | Non-systematic Assessment |
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| Vertigo | General disorders | Non-systematic Assessment |
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| Chronic kidney disease | General disorders | Non-systematic Assessment |
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| Chronic gingivitis | Infections and infestations | Non-systematic Assessment |
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| Ecthyma gangrenosum | Infections and infestations | Non-systematic Assessment |
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| Hepres zoster | Infections and infestations | Non-systematic Assessment |
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| Conjunctivitis | Infections and infestations | Non-systematic Assessment |
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| Otitis media | Infections and infestations | Non-systematic Assessment |
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| Rhinitis | Infections and infestations | Non-systematic Assessment |
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| Tinea corporis | Infections and infestations | Non-systematic Assessment |
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| Tinea infection | Infections and infestations | Non-systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | Non-systematic Assessment |
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| Urinary tract infection | Infections and infestations | Non-systematic Assessment |
|
| Alkaline phosphatase increased | Investigations | Non-systematic Assessment |
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| Systolic ejection murmur | Investigations | Non-systematic Assessment |
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| Anorexia | Metabolism and nutrition disorders | Non-systematic Assessment |
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| Hypokalemia | Metabolism and nutrition disorders | Non-systematic Assessment |
|
| Hyponatremia | Metabolism and nutrition disorders | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Hypertension | Vascular disorders | Non-systematic Assessment |
|
| Hypertension worsened | Vascular disorders | Non-systematic Assessment |
|
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.