Immunodeficiency in Cartilage-hair Hypoplasia: Correlation With Pulmonary Disease, Infections and Malignancy
试验速览
- 阶段
- 4 期
- 入组人数
- 5
- 主要终点
- Humoral response to vaccination
研究概览
简要总结
The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Any potential complication of VZV immunization, such as rash, will be discussed with the patients/caregivers beforehand and acyclovir will be used to treat any VZV related symptoms, consistent with the current practices. The investigators will verify the development of immune response to vaccination by testing for VZV antibodies and cell-mediated immunity.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •genetically confirmed cartilage-hair hypoplasia diagnosis
- •age >12 months
- •no history of chickenpox
- •informed concent of the patient/caregiver.
排除标准
- •positive serum IgG for varicella zoster virus
- •low CD4+ cell counts (<15% or <200 cells/mm3)
- •clinical or laboratory signs of severe immunodeficiency
- •ongoing intravenous or subcutaneous immunoglobulin treatment
结局指标
主要结局
Humoral response to vaccination
时间窗: 4-6 weeks post-vaccination
Cell-mediated response to vaccination
时间窗: 4-6 wks post-vaccination
Number and severity of adverse events to vaccination
时间窗: 0-60 days
次要结局
未报告次要终点
研究者
Paula Klemetti
MD, PhD, Consultant in pediatric gastroenterology and immunology
Helsinki University Central Hospital