Trial Outcomes & Findings for A Study Evaluating Epoetin Alfa 40,000 IU (International Units) Every Week or 80,000 IU Every Week Compared to Placebo in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes at Risk for Transfusion (NCT NCT00695396)
NCT ID: NCT00695396
Last Updated: 2012-10-05
Results Overview
Incidence of participants who received at least 1 Red Blood Cell (RBC) transfusion during the study (from randomization through the end of study)
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
25 participants
Primary outcome timeframe
Approximately 48 weeks
Results posted on
2012-10-05
Participant Flow
Participant milestones
| Measure |
Placebo
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Overall Study
STARTED
|
8
|
8
|
9
|
|
Overall Study
COMPLETED
|
1
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
7
|
8
|
8
|
Reasons for withdrawal
| Measure |
Placebo
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Overall Study
Study Closed Prematurely
|
5
|
6
|
8
|
|
Overall Study
Withdrawal by Subject
|
2
|
2
|
0
|
Baseline Characteristics
A Study Evaluating Epoetin Alfa 40,000 IU (International Units) Every Week or 80,000 IU Every Week Compared to Placebo in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes at Risk for Transfusion
Baseline characteristics by cohort
| Measure |
Placebo
n=8 Participants
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 Participants
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 Participants
(2 mL) subcutaneously once every week
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
21 Participants
n=4 Participants
|
|
Age Continuous
|
73 years
STANDARD_DEVIATION 10.52 • n=5 Participants
|
77.3 years
STANDARD_DEVIATION 9.78 • n=7 Participants
|
67.7 years
STANDARD_DEVIATION 9.89 • n=5 Participants
|
72.4 years
STANDARD_DEVIATION 10.44 • n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Region of Enrollment
CANADA
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Region of Enrollment
ITALY
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Region of Enrollment
RUSSIA
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
4 participants
n=5 Participants
|
6 participants
n=4 Participants
|
|
Region of Enrollment
USA
|
5 participants
n=5 Participants
|
7 participants
n=7 Participants
|
5 participants
n=5 Participants
|
17 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Approximately 48 weeksPopulation: The intent-to-treat (ITT) population was defined as all participants randomly assigned to a treatment group, regardless of whether they received any treatment.
Incidence of participants who received at least 1 Red Blood Cell (RBC) transfusion during the study (from randomization through the end of study)
Outcome measures
| Measure |
Placebo
n=8 Participants
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 Participants
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 Participants
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Red Blood Cell (RBC) Transfusion
|
5 participants
|
3 participants
|
1 participants
|
SECONDARY outcome
Timeframe: Day 29 through the end of study (approximately 48 weeks)Population: The intent-to-treat (ITT) population was defined as all participants randomly assigned to a treatment group, regardless of whether they received any treatment.
incidence of participants who received at least 1 RBC transfusion from Day 29 through the end of study (approximately 48 weeks).
Outcome measures
| Measure |
Placebo
n=8 Participants
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 Participants
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 Participants
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
RBC Transfusion From Day 29 Through the End of Study
|
4 participants
|
2 participants
|
1 participants
|
SECONDARY outcome
Timeframe: Approximately 48 weeksPopulation: The intent-to-treat (ITT) population.
Participants who were transfusion-dependent were those who received 4 or more RBC units during a consecutive 8-week period.
Outcome measures
| Measure |
Placebo
n=8 Participants
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 Participants
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 Participants
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Transfusion Dependent
|
2 participants
|
2 participants
|
1 participants
|
Adverse Events
Placebo
Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths
Epoetin Alfa 40000 IU
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Epoetin Alfa 80000 IU
Serious events: 4 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=8 participants at risk
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 participants at risk
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 participants at risk
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Aplasia Pure Red Cell
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Blood and lymphatic system disorders
Splenomegaly
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate Cancer
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Eye disorders
Vision Blurred
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Gastrointestinal Haemorrhage
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
General disorders
Asthenia
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Injury, poisoning and procedural complications
Humerus Fracture
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Injury, poisoning and procedural complications
Wrist Fracture
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
Other adverse events
| Measure |
Placebo
n=8 participants at risk
(1ml or 2 mL) subcutaneously once every week
|
Epoetin Alfa 40000 IU
n=8 participants at risk
(1 mL) subcutaneously once every week
|
Epoetin Alfa 80000 IU
n=9 participants at risk
(2 mL) subcutaneously once every week
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal Discomfort
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Haematochezia
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Oral Pain
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Rectal Haemorrhage
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/8
|
12.5%
1/8
|
22.2%
2/9
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
General disorders
Fatigue
|
25.0%
2/8
|
25.0%
2/8
|
11.1%
1/9
|
|
General disorders
Asthenia
|
12.5%
1/8
|
0.00%
0/8
|
22.2%
2/9
|
|
General disorders
Hyperthermia
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
General disorders
Oedema Peripheral
|
0.00%
0/8
|
12.5%
1/8
|
11.1%
1/9
|
|
General disorders
Pyrexia
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Blood and lymphatic system disorders
Anaemia
|
25.0%
2/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Blood and lymphatic system disorders
Leukopenia
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Blood and lymphatic system disorders
Neutropenia
|
12.5%
1/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Nervous system disorders
Dizziness
|
25.0%
2/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Nervous system disorders
Somnolence
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
25.0%
2/8
|
12.5%
1/8
|
11.1%
1/9
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea Exertional
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Respiratory, thoracic and mediastinal disorders
Productive Cough
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Injury, poisoning and procedural complications
Contusion
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Psychiatric disorders
Insomnia
|
12.5%
1/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Renal and urinary disorders
Pollakiuria
|
12.5%
1/8
|
0.00%
0/8
|
0.00%
0/9
|
|
Cardiac disorders
Palpitations
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
0.00%
0/8
|
25.0%
2/8
|
11.1%
1/9
|
|
Investigations
Blood Urine Present
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/8
|
0.00%
0/8
|
11.1%
1/9
|
|
Vascular disorders
Hypertension
|
0.00%
0/8
|
12.5%
1/8
|
0.00%
0/9
|
Additional Information
Senior Director, Head of Hematology and Nephrology
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place