A Randomized, Double Blind, Placebo Controlled, Multicenter Study Evaluating Epoetin Alfa Initiated at 40,000 IU Every Week or 80,000 IU Every Week Versus Placebo in Subjects With IPSS Low- or Intermediate-1 Risk Myelodysplastic Syndromes at Risk For Transfusion
Overview
- Phase
- Phase 3
- Intervention
- Epoetin alfa
- Conditions
- Myelodysplastic Syndromes
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Enrollment
- 25
- Primary Endpoint
- Red Blood Cell (RBC) Transfusion
- Status
- Terminated
- Last Updated
- 13 years ago
Overview
Brief Summary
The purpose of this study is to demonstrate that Epoetin alfa treatment reduces red blood cell transfusions in anemic patients with myelodysplastic syndromes (MDS). Myelodysplastic syndromes are a group of disorders characterized by progressive bone marrow failure and an increased risk of development of leukemia.
Detailed Description
This is a randomized (patients are assigned by chance to a treatment group), double-blind (neither the patient or the physician know which treatment is being received by the patient), placebo-controlled, multicenter study of epoetin alfa in anemic patients who are diagnosed with myelodysplastic syndromes (MDS) according to protocol-specified criteria. Patients meeting entry criteria for the study will be randomly assigned to receive epoetin alfa 40,000 IU or 80,000 IU or a matching volume of placebo administered by subcutaneous (under the skin) injection once every week. Doses of study drug will be withheld, decreased, or increased on the basis of weekly hemoglobin concentrations monitored in patients and predefined dose adjustment guidelines. An Independent Data Monitoring Committee (IDMC) will periodically review study data and for the assessment of disease progression, an independent central reviewer will review bone marrow specimens and peripheral blood counts. Safety will be monitored throughout the study at predetermined intervals and as clinically indicated by physical examination, laboratory tests and evaluation of adverse events. Patients in the Treatment Phase will be randomly assigned to receive once weekly epoetin alfa subcutaneously (SC) at a dose of 40,000 IU (1 mL) or 80,000 IU (2ML) or matching volume of placebo (1 mL or 2 mL) once every week for 48 weeks. Patients may continue to receive double-blinded treatment after 48-weeks.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of MDS according to protocol-specified criteria via bone marrow studies performed within 12 weeks before randomization
Exclusion Criteria
- •No prior or concurrent treatment with epoetin alfa or any other approved or experimental erythropoietin stimulating agents (ESAs) within the previous 12 months before randomization
- •No prior use of approved or experimental agents for the treatment of MDS or recent treatment with granulocyte colony stimulating factor (G-CSF) or granulocyte macrophage colony stimulating factor (GM-CSF) for the treatment of neutropenia
- •Patients must not have secondary MDS or anemia caused by factors other than MDS (including iron deficiency, vitamin B12 or folate deficiencies, hemolysis, chronic renal failure, or gastrointestinal bleeding)
- •No history (within 12 months) of deep venous thrombosis
- •or history (within 6 months) of stroke, acute coronary syndrome or other arterial thrombosis
- •Not currently receiving therapeutic anticoagulants or have uncontrolled hypertension
- •No uncontrolled disease or dysfunction deemed clinically significant by the Investigator not attributable to MDS
Arms & Interventions
001
Epoetin alfa 40 000 IU subcutaneously once every week (1 mL dose) for 48 weeks
Intervention: Epoetin alfa
002
Epoetin alfa 80 000 IU subcutaneously once every week (2 mL dose) for 48 weeks
Intervention: Epoetin alfa
003
Placebo Matching volume 1 mL for 48 weeks
Intervention: Placebo
004
Placebo Matching volume 2 mLfor 48 weeks
Intervention: Placebo
Outcomes
Primary Outcomes
Red Blood Cell (RBC) Transfusion
Time Frame: Approximately 48 weeks
Incidence of participants who received at least 1 Red Blood Cell (RBC) transfusion during the study (from randomization through the end of study)
Secondary Outcomes
- RBC Transfusion From Day 29 Through the End of Study(Day 29 through the end of study (approximately 48 weeks))
- Transfusion Dependent(Approximately 48 weeks)