Ignite Creation Date:
2025-12-25 @ 3:49 AM
Ignite Modification Date:
2025-12-26 @ 2:36 AM
Study NCT ID:
NCT00446602
Status:
WITHDRAWN
Last Update Posted:
2016-09-20
First Post:
2007-03-12
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Phase 2 Study to Evaluate the Safety and Effectiveness of Once Weekly or Once Every Two Week Dosing of Epoetin Alfa in Anemic Patients With Low- or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
Sponsor:
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Organization:
Study Overview
Official Title:
A Phase 2, Randomized, Open-Label Study To Assess The Safety And Efficacy Of Weekly (QW) Or Once Every Two Week (Q2W) Dosing Of Epoetin Alfa (PROCRIT) in Anemic Subjects With Low- or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
Status:
WITHDRAWN
Status Verified Date:
2011-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to company decision to focus resources on a larger, controlled study in this patient population.
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to evaluate the effectiveness of PROCRIT (Epoetin alfa) 80,000 Units given once weekly or 80,000 Units given once every two weeks in anemic patients with Low- or Intermediate-1 risk Myelodysplastic Syndromes (MDS).
Detailed Description:
This is a randomized (patients are assigned to a type of treatment by chance), open-label (both the patient and the physician know what treatment is being given), multi-center study in approximately 100 anemic patients with Low- or Intermediate-1 risk Myelodysplastic Syndromes (MDS). Patients with a diagnosis of MDS via bone marrow aspirate and biopsy according to World Health Organization (WHO) Criteria or French-American-British (FAB) Classification, and International Prognostic Scoring System (IPSS) of Low- or Intermediate-1 risk disease (\<=10% bone marrow blasts), a baseline hemoglobin (Hb) \< 10.0 g/dL \[defined as the average of at least 2 measurements (not influenced by red blood cell (RBC) transfusions for at least 1 week) greater than or equal to 1 week apart\], and who meet all other inclusion/exclusion criteria will be randomized to receive PROCRIT (Epoetin alfa) 80,000 Units under the skin (sc) once weekly (qw ) or 80,000 Units sc once every 2 weeks (q2w).
The total study duration is up to 30 weeks, including up to a 2-week screening phase, a 24-week dosing phase, a follow-up visit according to the patient's assigned visit schedule, and a 4-week safety follow-up phase.
Beginning at Week 13, and every week thereafter, patients will be assessed for Erythroid Response. Overall Erythroid Response (OER) (as per the 2000 International Working Group (IWG) Criteria) including Major and Minor Erythroid Responses is defined as: Major Erythroid Response: Having sustained one of the following criteria over a minimum of 8 weeks: \>2 g/dL rise in hemoglobin (Hb), OR transfusion independence for patients who were RBC transfusion dependent (defined as requiring 4 or more red blood cell (RBC) units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa) ) at baseline. Minor Erythroid Response is defined as: Having sustained one of the following criteria over a minimum of 8 weeks: 1-2 g/dL rise in Hb, OR 50 to \<100% transfusion reduction for patients who were RBC transfusion dependent (defined as requiring 4 or more RBC units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa)) at baseline.
Overall Erythroid Response (OER) (as per the 2006 Modified IWG Criteria) is one of the secondary endpoints of the study and is defined as: Having sustained one of the following criteria over a minimum of 8 weeks: \>= 1.5 g/dL rise in Hb, OR Reduction in transfusion requirements by at least 4 RBC units for patients who were RBC transfusion dependent (defined as requiring 4 or more RBC units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa) at baseline. Only RBC transfusions given for a Hb of \<= 9.0 g/dL pretreatment will count in the RBC transfusion response evaluation.
Fatigue assessments \[Brief Fatigue Inventory (BFI) and Medical Outcome Survey (MOS) Short Form-36 (SF-36)\] will be completed by patients at baseline (Day 1/Week 1), Week 9, Week 13, and at end of study. In addition, a Global Rating of Change in Level of Fatigue (single-item question) will be completed by patients at Week 13 and end of study.
Safety evaluations will be performed at specified intervals during the study. Hb, hematocrit (Hct), and blood pressure will be monitored weekly. Clinical safety will be assessed by the incidence and severity of adverse events, clinical laboratory tests (Hb and Hct), vital signs and physical examinations during the study period.
Patients will be randomized in a 1:1 ratio to receive PROCRIT (Epoetin alfa) 80,000 Units given under the skin once a week or 80,000 Units given under the skin once every 2 weeks. Dose adjustments will be made (i.e., dose increased or decreased, frequency decreased, or doses withheld) in response to Hb monitoring throughout the study and in order to maintain a Hb level in the target range of 11 to 12 g/dL.
The total study duration is up to 30 weeks, including up to a 2-week screening phase, a 24-week dosing phase, a follow-up visit according to the patient's assigned visit schedule, and a 4-week safety follow-up phase.
Beginning at Week 13, and every week thereafter, patients will be assessed for Erythroid Response. Overall Erythroid Response (OER) (as per the 2000 International Working Group (IWG) Criteria) including Major and Minor Erythroid Responses is defined as: Major Erythroid Response: Having sustained one of the following criteria over a minimum of 8 weeks: \>2 g/dL rise in hemoglobin (Hb), OR transfusion independence for patients who were RBC transfusion dependent (defined as requiring 4 or more red blood cell (RBC) units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa) ) at baseline. Minor Erythroid Response is defined as: Having sustained one of the following criteria over a minimum of 8 weeks: 1-2 g/dL rise in Hb, OR 50 to \<100% transfusion reduction for patients who were RBC transfusion dependent (defined as requiring 4 or more RBC units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa)) at baseline.
Overall Erythroid Response (OER) (as per the 2006 Modified IWG Criteria) is one of the secondary endpoints of the study and is defined as: Having sustained one of the following criteria over a minimum of 8 weeks: \>= 1.5 g/dL rise in Hb, OR Reduction in transfusion requirements by at least 4 RBC units for patients who were RBC transfusion dependent (defined as requiring 4 or more RBC units within 8 weeks prior to the first dose of PROCRIT (Epoetin alfa) at baseline. Only RBC transfusions given for a Hb of \<= 9.0 g/dL pretreatment will count in the RBC transfusion response evaluation.
Fatigue assessments \[Brief Fatigue Inventory (BFI) and Medical Outcome Survey (MOS) Short Form-36 (SF-36)\] will be completed by patients at baseline (Day 1/Week 1), Week 9, Week 13, and at end of study. In addition, a Global Rating of Change in Level of Fatigue (single-item question) will be completed by patients at Week 13 and end of study.
Safety evaluations will be performed at specified intervals during the study. Hb, hematocrit (Hct), and blood pressure will be monitored weekly. Clinical safety will be assessed by the incidence and severity of adverse events, clinical laboratory tests (Hb and Hct), vital signs and physical examinations during the study period.
Patients will be randomized in a 1:1 ratio to receive PROCRIT (Epoetin alfa) 80,000 Units given under the skin once a week or 80,000 Units given under the skin once every 2 weeks. Dose adjustments will be made (i.e., dose increased or decreased, frequency decreased, or doses withheld) in response to Hb monitoring throughout the study and in order to maintain a Hb level in the target range of 11 to 12 g/dL.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: