Efficacy and Safety of BT200 (Rondoraptivon 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
- Primary Outcome measure Platelet Counts
研究概览
简要总结
This randomized clinical trial with a cross-over design is being conducted at the Department of Clinical Pharmacology at the Medical University of Vienna, and a total of 4-6 patients with type 2B von Willebrand disease (VWD) will participate.
The main purpose of this clinical trial is to investigate the efficacy and safety of BT200, a new drug for thrombocytopenic patients with type 2B von Willebrand disease (VWD). Based on previous studies, we expect that this drug will inhibit the breakdown of von Willebrand factor (VWF) in small doses, leading to an increase in von Willebrand factor (VWF), platelet count, and factor VIII. This should also lead to a reduced tendency to bleed.
This study will begin with an observation phase and will then proceed in two periods of approximately 64 days each:
Placebo or BT200 will be administered subcutaneously at a dose of 12 mg on the first day of the study. After that, patients will self-administer the drug at a dose of 6 mg (0.4 mL) or placebo once a week for another 4 weeks starting the following week (a total of 4 times over a period of 4 weeks). During this time, they will be asked to come to our clinic for a follow-up visit.
After a "washout phase" lasting several weeks, during which patients do not receive the study drug/placebo but are asked to record any bleeding events, the second period begins on day 64:
BT200 or placebo is administered again, depending on what the patients received in the first period. Patients therefore receive the study drug for 4 weeks and placebo for 4 weeks; which is administered when is randomized; a follow-up examination also takes place during this period.
At the end of the second period, there is another "washout phase" lasting several weeks. On day 127, the final examination takes place at the clinic, after which patients have the opportunity to participate in an extension study (to be amended).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
placebo is non-distinguishable from verum based on physicochemical properties (colourless fluid)
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
- •≥18 years old
- •Type 2B VWD with thrombocytopenia and a recent bleeding history (e.g. recurrent haematomas)
- •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 protocol-related 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
研究组 & 干预措施
Placebo
Patients randomized to receive placebo first. They will receive the verum BT200 after an adequate washout period
干预措施: Placebo (Drug)
BT200
Patients randomized to receive BT200 (verum) first and placebo in the second phase after an adequate washout period.
干预措施: BT200 (Drug)
结局指标
主要结局
Primary Outcome measure Platelet Counts
时间窗: During the five-week Treatment Phase compared with the five-week Control Phase
Platelet Counts
Co-Primary Endpoint Clinically evident bleeding
时间窗: During the five-week Treatment Phase compared with the five-week Control Phase
number of clinically evident bleedings
次要结局
- von Willebrand factor antigen(During the five-week Treatment Phase compared with the five-week Control Phase)
- von Willebrand factor activity(During the five-week Treatment Phase compared with the five-week Control Phase)
- VWF activity collagen binding(During the five-week Treatment Phase compared with the five-week Control Phase)
- VWF:ristocetin co-factor activity(During the five-week Treatment Phase compared with the five-week Control Phase)
- Enzyme-linked immunosorbent assay (ELISA) for unbound VWF-A1 domain (REAADS® )(During the five-week Treatment Phase compared with the five-week Control Phase)
- Platelet function under high shear rates(During the five-week Treatment Phase compared with the five-week Control Phase)
- BT200 plasma concentrations(During the five-week Treatment Phase compared with the five-week Control Phase)
- Serious, drug-related AEs(During the five-week Treatment Phase compared with the five-week Control Phase)
- Premature terminations due to drug-related AEs(During the five-week Treatment Phase compared with the five-week Control Phase)
- Adverse events indicative of BT200 toxicity(During the five-week Treatment Phase compared with the five-week Control Phase)
研究者
Christian Schoergenhofer
Principal Investigator
Medical University of Vienna