Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Proportion of HCPs providing the correct response
研究概览
简要总结
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in hematopoietic cell lineages, thereby preventing impaired immune function. STRIMVELIS is indicated for the treatment of patients with ADA- severe combined immunodeficiency (SCID), for whom suitable human leukocyte antigen (HLA)-matched related stem cell donor is not available. The objective of this study is to evaluate the effectiveness of routine and additional risk minimization measures by assessing the understanding of referring health care providers (HCPs) and parents/carers (hereby referred as participants) with regard to the specific risks associated with STRIMVELIS. In this cross-sectional study, surveys will be provided to referring HCPs and parents/carers of children approximately six months after treatment with STRIMVELIS. The study will recruit for approximately two years or until a maximum of 10 referring HCPs and 10 parents/carers have completed their respective surveys, whichever occurs first.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •HCPs or HCPs' close family members may not have been employees of Orchard, Pharmaceutical Product Development, LLC (PPD), the Food and Drug Administration (FDA), or the European Medicines Agency (EMA).
- •HCPs must be licensed
- •An HCP must not have previously completed a survey regarding STRIMVELIS educational materials.
- •An HCP must have previously referred a patient for STRIMVELIS treatment.
- •Parents/carers or parents'/carers' close family members may not have been employees of Orchard, PPD, FDA, or EMA.
- •A parent/carer must not have previously completed a survey regarding STRIMVELIS educational materials.
- •A parent's or carer's child must have previously received treatment with STRIMVELIS
排除标准
- •No exclusion criteria
研究组 & 干预措施
Health care providers
A HCP survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS summary of product characteristics and educational materials
干预措施: STRIMVELIS (Drug)
Parent/carer
A parent/carer survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS Patient Information Leaflet and educational materials
干预措施: STRIMVELIS (Drug)
结局指标
主要结局
Proportion of HCPs providing the correct response
时间窗: Up to 2 years
A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.
Proportion of Parents/Carers providing the correct response
时间窗: Up to 2 years
A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.
次要结局
未报告次要终点