Trial Outcomes & Findings for A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes. (NCT NCT00551291)
NCT ID: NCT00551291
Last Updated: 2016-07-25
Results Overview
International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) \<11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Up to approximately 2 years
Results posted on
2016-07-25
Participant Flow
Participant milestones
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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|---|---|
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Overall Study
STARTED
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10
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Overall Study
COMPLETED
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9
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Overall Study
NOT COMPLETED
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1
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Reasons for withdrawal
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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|---|---|
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Overall Study
Physician Decision
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1
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Baseline Characteristics
A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.
Baseline characteristics by cohort
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 Participants
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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|---|---|
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Age, Continuous
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75.00 years
STANDARD_DEVIATION 5.17 • n=5 Participants
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Sex: Female, Male
Female
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3 Participants
n=5 Participants
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Sex: Female, Male
Male
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7 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Up to approximately 2 yearsInternational Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) \<11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment.
Outcome measures
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 Participants
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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|---|---|
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Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
Week 18 (n=7)
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71.43 percentage of participants
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Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
End of study (n=3)
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100.00 percentage of participants
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Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
Week 12 (n=4)
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50.00 percentage of participants
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PRIMARY outcome
Timeframe: Up to approximately 2 yearsOutcome measures
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 Participants
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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Mean Number of Blood Transfusions Per Visit
Week 12 (n=6)
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5.83 transfusions/visit
Standard Deviation 2.86
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Mean Number of Blood Transfusions Per Visit
Week 18 (n=5)
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2.80 transfusions/visit
Standard Deviation 1.92
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Mean Number of Blood Transfusions Per Visit
Baseline (n=8)
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4.13 transfusions/visit
Standard Deviation 2.30
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Mean Number of Blood Transfusions Per Visit
End of Study (n=3)
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2.33 transfusions/visit
Standard Deviation 1.53
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SECONDARY outcome
Timeframe: Up to approximately 2 yearsAn AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.
Outcome measures
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 Participants
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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Percentage of Participants With at Least One Adverse Event (AE)
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90.00 percentage of participants
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Adverse Events
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
Serious events: 4 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 participants at risk
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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Infections and infestations
Pneumonia
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20.0%
2/10 • Up to approximately 2 years.
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Infections and infestations
Bronchitis
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10.0%
1/10 • Up to approximately 2 years.
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Cardiac disorders
Cardiac insufficiency
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10.0%
1/10 • Up to approximately 2 years.
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Hepatobiliary disorders
Colecistitis
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10.0%
1/10 • Up to approximately 2 years.
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Musculoskeletal and connective tissue disorders
Achilles tendon´s break
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10.0%
1/10 • Up to approximately 2 years.
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Gastrointestinal disorders
Rectal bleeding
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10.0%
1/10 • Up to approximately 2 years.
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Other adverse events
| Measure |
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta
n=10 participants at risk
Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate mofetil: 1 gm twice daily orally until end of study.
Prednisone: 10 mg/day orally until end of study.
Erythropoietin Beta: Recombinant human erythropoietin beta at doses of 30,000 IU/week by the subcutaneous route for 6 weeks.
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|---|---|
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Infections and infestations
Upper respiratory tract catarrh
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30.0%
3/10 • Up to approximately 2 years.
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Infections and infestations
Respiratory infection
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10.0%
1/10 • Up to approximately 2 years.
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Infections and infestations
Bronchial infection
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10.0%
1/10 • Up to approximately 2 years.
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Infections and infestations
Influenza
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10.0%
1/10 • Up to approximately 2 years.
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Infections and infestations
Herpes labialis
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10.0%
1/10 • Up to approximately 2 years.
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Gastrointestinal disorders
Diarrhea
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20.0%
2/10 • Up to approximately 2 years.
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Gastrointestinal disorders
Gastroenteritis
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10.0%
1/10 • Up to approximately 2 years.
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Gastrointestinal disorders
Epigastralgia
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10.0%
1/10 • Up to approximately 2 years.
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Cardiac disorders
Dyspnea on exertion
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10.0%
1/10 • Up to approximately 2 years.
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Cardiac disorders
Dyspnea to great efforts
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10.0%
1/10 • Up to approximately 2 years.
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Cardiac disorders
Palpitations
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10.0%
1/10 • Up to approximately 2 years.
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Musculoskeletal and connective tissue disorders
Achilles tendon´s tendinitis
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10.0%
1/10 • Up to approximately 2 years.
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Musculoskeletal and connective tissue disorders
Coxalgia
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10.0%
1/10 • Up to approximately 2 years.
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General disorders
Asthenia
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20.0%
2/10 • Up to approximately 2 years.
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General disorders
Dysthermia feeling
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10.0%
1/10 • Up to approximately 2 years.
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Skin and subcutaneous tissue disorders
Facial allergic reaction
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10.0%
1/10 • Up to approximately 2 years.
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Respiratory, thoracic and mediastinal disorders
Occasional tightness in the chest
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10.0%
1/10 • Up to approximately 2 years.
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Psychiatric disorders
Nervousness
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10.0%
1/10 • Up to approximately 2 years.
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Injury, poisoning and procedural complications
Chest injury due to fall
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10.0%
1/10 • Up to approximately 2 years.
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Endocrine disorders
Decompensated diabetes mellitus
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10.0%
1/10 • Up to approximately 2 years.
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Blood and lymphatic system disorders
Splenomegaly increase
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10.0%
1/10 • Up to approximately 2 years.
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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