The Global Angelman Syndrome Registry
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 5,000
- 试验地点
- 2
- 主要终点
- Gather longitudinal data on individuals living with Angelman Syndrome
研究概览
简要总结
The Global Angelman Syndrome Registry is an online patient organisation driven registry to collect information about the natural history of children and adults with Angelman Syndrome. The registry will facilitate 1) recruitment for clinical trials into therapies and interventions to benefit participants with Angelman Syndrome and their families, and 2) advancement of research and best standards of care for Angelman Syndrome.
The registry is currently available in English, Spanish, Traditional Chinese, Italian, Polish, Hindi, and Brazilian Portuguese.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Angelman Syndrome
排除标准
- 未提供
研究组 & 干预措施
Individuals with Angelman Syndrome
Individuals from birth to adulthood with Angelman Syndrome
干预措施: Observational study only (Other)
结局指标
主要结局
Gather longitudinal data on individuals living with Angelman Syndrome
时间窗: 70 years (lifespan)
Parent/ caregiver reporting on diagnosis, clinical status, and patient-reported outcomes of individual living with Angelman Syndrome. This will be achieved by inviting parents/ caregivers with additional questionnaire like modules, and tracking changes in their responses over time.
次要结局
未报告次要终点
研究者
Associate Professor Helen (Honey) Heussler
Associate Professor, Paediatrics and Child Health
The University of Queensland