The purpose of this study is to determine the efficacy and safety of rhIL-11, when given subcutaneously for six consecutive months, in reducing menstrual blood loss in women with type 1 von Willebrand disease and refractory menorrhagia. Efficacy will be measured by subjective bleeding severity scale and pictorial bleeding chart. Safety will be measured by the frequency of adverse events, including fever, headache, fatigue, myalgias, arthralgias, fluid retention, or edema.
Primary Outcome Measures:
- The primary outcome measure is subjective estimate of blood loss during six menstrual cycles. [ Time Frame: The time frame is up to 7 months per subject ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- A secondary outcome measure is the mechanism of IL-11 effect by VWFmRNA. [ Time Frame: The time frame is up to 7 months per subject. ] [ Designated as safety issue: No ]
- A secondary outcome measure is the frequency of IL-11 associated adverse events. [ Time Frame: The time frame is up to 7 months per subject. ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: |
10 |
| Study Start Date: |
January 2008 |
| Estimated Study Completion Date: |
December 2010 |
| Estimated Primary Completion Date: |
December 2010 (Final data collection date for primary outcome measure) |
|
A: Experimental
|
Drug: Neumega (Oprelvekin, Interleukin 11, IL-11)
25 micrograms/kg by subcutaneous injection once daily for four days, then once daily on day 1-7 during each of six consecutive menstrual cycles
|
This study is a prospective, single-center Phase II trial of Neumega (rhIL-11) in women with type 1 VWD and menorrhagia refractory to estrogen, hormones, or hemostatic agents. It is anticipated that 10 subjects who meet eligibility criteria will enroll and complete this study. All aspects of this study, including the rhIL-11 injections and the screening, hemostatic and safety monitoring, and coagulation testing, are considered experimental. The specific objectives are to determine the efficacy and safety of rhIL-11 in reducing menstrual blood loss in adult women with type 1 VWD during six consecutive menstrual cycles, and to determine the mechanism of the hemostatic response of rhIL-11.
Purpose
