A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Patient/Parent Global Assessment of Disease Activity
研究概览
简要总结
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Clinical trials are currently examining its efficacy in other diseases associated with elevations of these cytokines, including NLRC4-associated disease and hidradenitis suppurativa. This study proposes to assess the effectiveness of MAS825 in a single patient with XLP-2, who has previously demonstrated response to blockade of IL-1 beta and IL-18. Given the lack of alternative pharmaceutical options for XLP-2, this represents the only known medication option that avoids the toxicity associated with high-dose corticosteroids and the morbidity associated with hematopoietic stem cell transplantation.
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
- 13 Years 至 14 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Known patient with XIAP genetic defect under the care of the investigator
排除标准
- 未提供
研究组 & 干预措施
Active treatment
干预措施: MAS825 (Drug)
结局指标
主要结局
Patient/Parent Global Assessment of Disease Activity
时间窗: 5 years
Likert Scale: 0 = Absent (no) disease associated clinical signs and symptoms 1. = Minimal disease associated signs and symptoms 2. = Mild disease associated signs and symptoms 3. = Moderate disease associated signs and symptoms 4. = Severe disease associated signs and symptoms
Impact of MAS825 on Physician Global Assessment Score
时间窗: 5 years
Likert Scale: 0 = Absent (no) disease associated clinical signs and symptoms 1. = Minimal disease associated signs and symptoms 2. = Mild disease associated signs and symptoms 3. = Moderate disease associated signs and symptoms 4. = Severe disease associated signs and symptoms
Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms
时间窗: 5 years
The signs and symptoms assessed include: 1) Abdominal pain 2) Diarrhea 3) Fever using a Likert scale: 0 = Absent 1. = Minimal 2. = Mild 3. = Moderate 4. = Severe
次要结局
未报告次要终点
研究者
Dilan Dissanayake
Project Investigator
The Hospital for Sick Children