An Observational Study of VYKAT™ XR (Diazoxide Choline Extended-Release Tablets) in Patients With Prader-Willi Syndrome (PWS)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 200
- 试验地点
- 33
- 主要终点
- Assess safety profile of patients with PWS treated with VYKAT XR.
研究概览
简要总结
Prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with PWS treated with VYKAT XR.
详细描述
Patients with PWS at participating sites who are initiating treatment or have initiated treatment with VYKAT XR, including patients who were previously treated with DCCR in a prior clinical study, will be invited to participate. Data from treatment prior to enrollment either from clinical practice or from previous DCCR clinical trials may be collected retrospectively.
Clinical care will not be mandated by the protocol.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients currently receiving or initiating treatment with VYKAT XR, including patients who previously received DCCR or are transitioning from DCCR to VYKAT XR in the context of a prior DCCR clinical trial.
- •Patients and/or caregivers must provide written informed consent.
排除标准
- 未提供
研究组 & 干预措施
Patients with PWS who are currently receiving or initiating treatment with VYKAT XR.
All patients with PWS at participating sites who are initiating treatment or have initiated treatment with VYKAT XR at the time of enrollment into this observational study, including patients who were previously treated with DCCR in a prior clinical study, will be invited to participate. Although the overall observational study is prospective, data from treatment prior to enrollment into the observational study, either from clinical practice or from DCCR clinical trials, may be collected retrospectively.
干预措施: VYKAT XR (Drug)
结局指标
主要结局
Assess safety profile of patients with PWS treated with VYKAT XR.
时间窗: Adverse events for each patient will be collected from the time of first dose on VYKAT XR until the last contact with the patient.
The safety profile will be based on adverse events, including nonserious adverse events, serious adverse events, and adverse events leading to discontinuation of VYKAT XR.
次要结局
未报告次要终点