Phase II Comparison Study of Hemostatic Efficacy of Escalating Doses of Interleukin-11 (rhIL-11, Neumega) in Subjects With Type 1 Von Willebrand Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- The number and percent increase of VWD coagulation tests after seven daily doses of rhIL-11, boosted by DDAVP day 7.
研究概览
简要总结
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate:
- if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal
- if rhIL-11 and DDAVP together will boost VWF levels even higher
- the onset, peak, and duration of rhIL-11 effect
- if rhIL-11 is safe in individuals with Von Willebrand Disease
详细描述
This is a prospective, single center, open-label, escalating dose Phase II comparison study of interleukin-11 (rhIL-11, Neumega) in subjects with type 1 Von Willebrand Disease (VWD).
The purpose is to establish the clinical safety and hemostatic efficacy of rhIL-11 in individuals with type 1 Von Willebrand disease.
Study subjects will include the following subjects:
- age >= 18 years of age
- diagnosis of VWD confirmed by: 2a) at least 2 of 4 abnormal vWD-related coagulation tests; 2b) a past bleeding history
A total of 10-16 subjects are anticipated to be enrolled and complete the study. The specific aims of the study are:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females 18 years of age or older
- •Confirmed VWD by 2 of 4 VWD coagulation tests abnormal
- •A past bleeding history
- •No hormone, oral contraceptive, estrogen use in past 8 weeks
- •Willingness to have blood drawn
- •Willingness to sign informed consent
排除标准
- •Presence of other bleeding disorder, e.g. acquired VWD, thrombocytopenia
- •Use of estrogens, hormones, oral contraceptives in past 8 weeks
- •Use of immunomodulatory or experimental drugs or diuretics
- •Pregnant or lactating women
- •Past 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 FVIII, VWF within 5 days of study
研究组 & 干预措施
A
rhIL-11 (Interleukin-11, Neumega) 25 mcg/kg subcutaneously daily for 7 days
干预措施: recombinant interleukin-11 (Drug)
B
rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days
干预措施: recombinant interleukin-11 (Drug)
C
rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days
干预措施: recombinant interleukin-11 (Drug)
结局指标
主要结局
The number and percent increase of VWD coagulation tests after seven daily doses of rhIL-11, boosted by DDAVP day 7.
时间窗: The time frame is up to 14 days per subject.
次要结局
- The number and frequency of IL-11 associated adverse events.(The time frame is up to 14 days per subject.)
- The mechanism of IL-11 biologic effect by VWFmRNA.(The time frame is within 14 days per subject.)
研究者
Margaret Ragni
Professor of Medicine
University of Pittsburgh