VIrtual STudy in Achondroplasia for the US (VISTA)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 170
- 试验地点
- 11
- 主要终点
- Height
研究概览
简要总结
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor.
Data will be collected in two formats for the primary pediatric study population:
- Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. .
- Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data.
Data will be collected in the following format for the adult cohort:
- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care.
The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment.
The secondary study population will include 20 adolescent and adult participants.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 0 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Physician diagnosis of achondroplasia
- •Age at time of enrollment:
- •Primary Pediatric Cohort: ≤13 years old Secondary Adolescent and Adult Cohort: ≥14 years old
- •Receiving medical care in the United States
- •Complete PicnicHealth's onboarding process, including signing informed consent and authorization for medical record retrieval
排除标准
- •Lack of any medical records
结局指标
主要结局
Height
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
sitting and standing measured in centimetres
Annualized growth velocity (AGV)
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in centimeters/year
Weight
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in kilograms
Body Mass Index
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in kg/m2
Changes in health-related quality of life (HRQoL)
时间窗: every 6 months starting from time of study enrolment up to 5 years
assessed using the Pediatric Quality of Life Inventory (PedsQL)
Head Circumference
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in centimeters
Medical diagnoses by age
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Clinical examination and/or radiological assessment
Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed
时间窗: every 6 months starting from time of study enrolment up to 5 years
assessed using a medication adherence questionnaire to capture the number of missed doses in the prior month. Parent(ObsRO) version for all participants enrolled ages 5-13 years
Surgical procedures and/or medical interventions by age
时间窗: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Changes in physical functioning
时间窗: every 6 months starting from time of study enrolment up to 5 years
assessed using the Mobility and Upper Extremity Patient-Reported Outcomes Measurement Information System (PROMIS) tools
次要结局
未报告次要终点