Trial Outcomes & Findings for A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS) (NCT NCT02145026)
NCT ID: NCT02145026
Last Updated: 2020-04-24
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
100 participants
Primary outcome timeframe
Week 12
Results posted on
2020-04-24
Participant Flow
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
100
|
|
Overall Study
COMPLETED
|
88
|
|
Overall Study
NOT COMPLETED
|
12
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Death
|
4
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Physician Decision
|
4
|
Baseline Characteristics
A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)
Baseline characteristics by cohort
| Measure |
Epoetin Beta
n=99 Participants
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.
|
|---|---|
|
Age, Continuous
|
72.9 Years
STANDARD_DEVIATION 10.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
69 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
99 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
99 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 12Population: 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.
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.
Outcome measures
| Measure |
Epoetin Beta
n=99 Participants
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.
|
|---|---|
|
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria
|
60 Participants
|
SECONDARY outcome
Timeframe: Week 12Population: 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.
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.
Outcome measures
| Measure |
Epoetin Beta
n=99 Participants
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.
|
|---|---|
|
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 Percentage of Participants
|
SECONDARY outcome
Timeframe: Week 12Population: 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.
Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of \>0.5x10\^9/L.
Outcome measures
| Measure |
Epoetin Beta
n=99 Participants
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.
|
|---|---|
|
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 Percentage of Participants
|
SECONDARY outcome
Timeframe: From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)Population: The safety population included all participants that received at least one dose of study medication.
Outcome measures
| Measure |
Epoetin Beta
n=99 Participants
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.
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|---|---|
|
Percentage of Participants With Adverse Events
|
48.48 Percentage of Participants
|
Adverse Events
Epoetin Beta
Serious events: 21 serious events
Other events: 48 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Epoetin Beta
n=99 participants at risk
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.
|
|---|---|
|
Blood and lymphatic system disorders
Autoimmune haemolytic anaemia
|
1.0%
1/99 • Number of events 2 • Approximately 56 months
|
|
Cardiac disorders
Congestive heart failure
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Cardiac disorders
Cardiac tamponade
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Cardiac disorders
Cardiogenic shock
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Cardiac disorders
Tachyarrhythmia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Gastrointestinal bleeding
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Esophageal varices
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Abscess oral
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Hospital acquired pneumonia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Lobar pneumonia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Necrotizing fasciitis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Pneumonia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Sepsis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Septic shock
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Syphilis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Recurrent urinary tract infection
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Urinary tract infection
|
1.0%
1/99 • Number of events 2 • Approximately 56 months
|
|
Infections and infestations
Urosepsis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
1.0%
1/99 • Number of events 2 • Approximately 56 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Renal and urinary disorders
Acute kidney injury
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
1.0%
1/99 • Number of events 3 • Approximately 56 months
|
|
Vascular disorders
Hypertension worsened
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Vascular disorders
Hypertensive emergency
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
Other adverse events
| Measure |
Epoetin Beta
n=99 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Acute diarrhea
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Aphthous ulcer
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Constipation
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Nausea
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Gastrointestinal disorders
Short-bowel syndrome
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Blood and lymphatic system disorders
Anemia
|
2.0%
2/99 • Number of events 2 • Approximately 56 months
|
|
Blood and lymphatic system disorders
Eosinophilia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Blood and lymphatic system disorders
Anemia aggravated
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Anxiety
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Leg pain
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Lymphatic obstruction
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Closed fracture
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Elevated blood pressure
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Fatigue
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Gallstones
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Headache
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Increased blood pressure
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Uric acid level increased
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Insomnia
|
3.0%
3/99 • Number of events 3 • Approximately 56 months
|
|
General disorders
Leg edema
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Low back pain
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Epistaxis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Rash
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Purpura senile
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Vertigo
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
General disorders
Chronic kidney disease
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Chronic gingivitis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Ecthyma gangrenosum
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Hepres zoster
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Conjunctivitis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Otitis media
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Rhinitis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Tinea corporis
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Tinea infection
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Infections and infestations
Upper respiratory tract infection
|
3.0%
3/99 • Number of events 3 • Approximately 56 months
|
|
Infections and infestations
Urinary tract infection
|
2.0%
2/99 • Number of events 2 • Approximately 56 months
|
|
Investigations
Alkaline phosphatase increased
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Investigations
Systolic ejection murmur
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Metabolism and nutrition disorders
Anorexia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.0%
2/99 • Number of events 2 • Approximately 56 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.0%
1/99 • Number of events 1 • Approximately 56 months
|
|
Vascular disorders
Hypertension
|
6.1%
6/99 • Number of events 6 • Approximately 56 months
|
|
Vascular disorders
Hypertension worsened
|
2.0%
2/99 • Number of events 2 • Approximately 56 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER