Early Intervention With Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome (TMA/aHUS)-Associated Multiple Organ Dysfunction Syndrome (MODS) in Hematopoietic Stem Cell Transplant (HCT) Recipients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 3
- 主要终点
- Survival
研究概览
简要总结
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.
详细描述
This clinical trial is a prospective single arm multi-institution study in children and young adults undergoing allogeneic or autologous hematopoietic stem cell transplantation who will receive early therapy with eculizumab to prevent TMA-associated MODS after transplantation. The purpose of this research study is to examine efficacy of complement blocker eculizumab in HCT recipients with high risk TMA and to determine optimal eculizumab dosing regimen for HCT recipients with TMA using PK/PD studies. All patients will receive therapy based on their weight for 24 weeks. Survival will be assessed at 6 months from TMA diagnosis.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age undergoing allogeneic or autologous HCT
- •Histologic TMA diagnosis OR clinical TMA diagnosis and presenting with high risk disease features including elevated plasma sC5b-9 above laboratory normal value (≥244ng/ml) and proteinuria measured as ≥30mg/dL of protein on random urinalysis x2 or protein/creatinine ratio ≥1mg/mg or patient receiving renal replacement therapy.
- •Minimum weight of ≥ 5kg.
排除标准
- •Known hypersensitivity to any constituent of the study medication.
- •Subjects with unresolved serious Neisseria meningitides infection or progressive severe infection.
- •Patients with diagnosis of TTP as defined by ADAMST13 activity test <10%.
- •Patients previously treated with eculizumab or other complement blocker for TMA within the 60 days prior to first dose of study treatment.
研究组 & 干预措施
Eculizumab
All patients will receive eculizumab based on their weight for 24 weeks.
干预措施: Eculizumab (Drug)
结局指标
主要结局
Survival
时间窗: 6 months
Survival at 6 months after the date of TMA diagnosis
次要结局
- Number of Participants With Organ Dysfunction(1 year)
- Non-relapse Mortality(1 year)