Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 10
- 试验地点
- 9
- 主要终点
- Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
研究概览
简要总结
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
详细描述
This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days.
Part A: dose algorithm, safety and efficacy
Part B: safety and efficacy
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
- •Undergone allogeneic or autologous HSCT.
- •TMA diagnosis within a year of their allogeneic or autologous HSCT.
- •Clinical or histological diagnosis of TMA
- •Provision of written informed consent.
- •Provision of informed assent
排除标准
- •Patients weighing less than 5 kg.
- •Patients with a positive direct Coombs' test.
- •Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
- •Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
- •Grade 4 Acute graft-versus-host disease (GVHD)
- •Received eculizumab or any other complement blocker therapy at any time.
- •Known hypersensitivity to the active ingredient or excipients
- •If an enrolled patient has a positive ADAMTS13 test (<10%) returned from their screening assessment, the patient should be withdrawn from the study
研究组 & 干预措施
nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and develop transplant-associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT
干预措施: nomacopan (rVA576) (Drug)
结局指标
主要结局
Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
时间窗: 24 weeks
Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24 Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
次要结局
- Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)(24 weeks)
- Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)(24 weeks)
- Normalisation of Lab Parameters(24 weeks)
- Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.(24 weeks)