A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores
研究概览
简要总结
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.
详细描述
Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized.
This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.
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
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 18 years old
- •Clinical diagnosis of pediatric-onset HPP
- •Naïve to asfotase alfa
- •Expected to begin treatment with asfotase alfa for HPP
- •Registered in OneSource
- •Willing and able to provide voluntary, verbal informed consent to participate in this study
排除标准
- •Pregnant or breastfeeding
- •Unable to speak and understand English
- •Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points
研究组 & 干预措施
Participants with Pediatric-onset HPP
Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.
干预措施: asfotase alfa (Drug)
结局指标
主要结局
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores
时间窗: Baseline, up to 12 months
次要结局
未报告次要终点