Phase II Clinical Efficacy Trial of Recombinant Interleukin-11 (rhIL-11, Neumega) in Women With Type 1 Von Willebrand Disease and Refractory Menorrhagia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- >50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Females 18-45 years of age
- •Confirmed type 1 VWD, as defined by low VWF:RCoF or low VWF:Ag and qualitatively normal VWF multimers
- •Menorrhagia refractory to estrogens, hormones, hemostatic agents
- •Willingness to have blood drawn
排除标准
- •Use of immunomodulatory or experimental drugs, or diuretics
- •Pregnant or lactating women or those unwilling to use contraception during study
- •Previous cardiac disease, congestive failure, arrhythmia (e.g., atrial fibrillation, atrial flutter), hypertension, MI, stroke, or thrombosis
- •Past allergic reaction to Neumega or DDAVP
- •Surgery within the past 8 weeks
- •Inability to comply with study protocol requirements
- •Concomitant use of antiplatelet drugs, anticoagulants, dextran, aspirin or NSAIDs
- •Treatment with DDAVP, cryoprecipitate, whole blood, plasma and plasma derivatives containing substantial quantities of FVIII and/or VWF within five days of study
研究组 & 干预措施
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
干预措施: Oprelvekin, Interleukin 11, IL-11 (Drug)
结局指标
主要结局
>50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months
时间窗: 6 months
Subjective estimate of blood loss was measured by using a Pictorial blood assessment chart (PBAC) . The PBAC measures scores on a scale where 0 to 100 is normal and greater than 100-200 are abnormal.
次要结局
- No. of Subjects With IL-11 Associated Adverse Events.(The time frame is up to 7 months per subject.)
- No. of Subjects With Detectable VWF mRNA (Von Willebrand Factor Messenger RNA).(The time frame is up to 7 months per subject.)
研究者
Margaret Ragni
Professor
University of Pittsburgh