Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 发起方
- 主要终点
- A composite score of clinical and biochemical signs of disease activity
研究概览
简要总结
Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to determine why some patients need more frequent infusions of plasma than others to prevent acute attacks of the disease.
详细描述
Patients with congenital TTP have an inherited lack of function or amount of a protein in plasma called ADAMTS13, that otherwise is responsible for cleaving large von Willebrand-molecules into smaller parts. The patients suffer recurrent attacks of clotting of small blood vessels, that can cause damage to major organs, including the central nervous system. Acute attacks can be treated successfully with infusions of human plasma, and some patients also receive regular plasma therapy for prevention of acute attacks. A small group of patients receive preventive plasma infusions twice or more weekly, indicating a much higher need for plasma than what is otherwise recommended for preventive therapy. Do these patients have an ongoing activity of their disease despite a stimulus? Or a higher turn-over of transfused ADAMTS13? Have these patients developed antibodies against transfused ADAMTS13? Are any symptoms correlated with signs of disease activity?
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of congenital TTP
- •On regular prophylactic plasma treatment: > Once weekly (group 1) or >1 <3 times monthly (group 2)
- •Between 18 and 65 years
- •Capable of understanding and complying with the protocol
排除标准
- •Pregnancy
- •Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period
研究组 & 干预措施
Octaplas infusion and placebo (group 1)
Active treatment with randomly assigned 400 ml octaplas intravenously 2-3 times a week and 400 ml placebo (for octaplas)intravenously 2-3 times a week over two weeks.
干预措施: Octaplas infusion and placebo (group 1) (Drug)
Octaplas infusion and placebo (group 2)
Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks
干预措施: Octaplas infusion and placebo (group 2) (Drug)
结局指标
主要结局
A composite score of clinical and biochemical signs of disease activity
时间窗: 4 hours and 24 hours after intervention
Biochemical signs of disease activity are scored by measurements of lactate dehydrogenase, hemoglobin, platelet counts and ADAMTS13 activity. Clinical signs of disease activity are measured by an evaluator's score and subjects symptom screening
次要结局
- ADAMTS13 activity half-life(4 hours and 24 hours after intervention)
- ADAMTS13 allo-antibodies(At baseline)