VIrtual STudy in Achondroplasia for the US (VISTA)

Condition(s) studied

Study summary

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:

1. 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. .
2. 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.

Eligibility

Primary outcome measure(s)

• Height — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  sitting and standing measured in centimetres
• Weight — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  measured in kilograms
• Body Mass Index — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  measured in kg/m2
• Head Circumference — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  measured in centimeters
• Annualized growth velocity (AGV) — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  measured in centimeters/year
• Medical diagnoses by age — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
  Clinical examination and/or radiological assessment
• Surgical procedures and/or medical interventions by age — Retrospective data and prospective data for up to 5 years starting from time of study enrolment
• Changes in physical functioning — 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
• Changes in health-related quality of life (HRQoL) — every 6 months starting from time of study enrolment up to 5 years
  assessed using the Pediatric Quality of Life Inventory (PedsQL)
• Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed — 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

Trial sites (8)