2024-518294-34-01招募中2 期
Efficacy and Safety of BT200 (rondaptivon pegol) in Patients with Type 2B von Willebrand disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Co-Primary endpoints: Platelet counts, number of monthly bleedings events
研究概览
简要总结
To examine the effects of BT200 on platelet count, VWF indices, FVIII and bleeding in type 2B VWD
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Type 2B VWD with thrombocytopenia and a recent bleeding history
- •repeated thrombocytopenia in medical history
- •≥18 years of age
- •Able to comprehend and to give informed consent
- •Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocolrelated procedures
排除标准
- •Clinically significant medical history or ongoing chronic illness that would jeopardise the safety of the patient or compromise the quality of the data derived from his/her participation in this study
- •History of significant drug allergy or anaphylactic reactions
- •Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the patient to be able to comply fully with study procedures
- •Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the patient's welfare or the integrity of the study's results
- •Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start
结局指标
主要结局
Co-Primary endpoints: Platelet counts, number of monthly bleedings events
Co-Primary endpoints: Platelet counts, number of monthly bleedings events
次要结局
- Pharmacokinetics: • BT200 concentrations (and derived PK parameters) • Half-life of the substituted Factor VIII product used with and without BT200 (pop-PK-sub-study)
- Pharmacodynamics: • VWF antigen (VWF Ag) • VWF:ristocetin co-factor assay (VWF:RCo) • VWF activity (VWF:GpIbM assay) • VWF collagen binding assay (VWF:CBA) • Enzyme-linked immunosorbent assay (ELISA) for unbound VWF A1 domain (REAADS®) • Whole blood ristocetin induced platelet aggregation (Multiplate®) • Collagen Adenosine Diphosphate closure times measured by the platelet function analyser (PFA-100®)
研究者
Department of Clinical Pharmacology
Scientific
Medical University Of Vienna
研究点 (1)
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