VIrtual STudy in Achondroplasia for the US (VISTA)

Launched by BIOMARIN PHARMACEUTICAL · Dec 10, 2023

Keywords

ClinConnect Summary

This clinical trial is studying children with achondroplasia, a genetic condition that affects bone growth and leads to short stature. The study aims to gather information about the experiences and treatments of these children, including those who are being treated with a medication called VOXZOGO™ and those who are not. Enrollment for the study began in February 2023 and is expected to last about five years, but it might be extended. Data will be collected through participants' health records and through surveys about their health and treatment outcomes.

To be eligible for the study, children must have a doctor’s diagnosis of achondroplasia, be receiving care in the United States, and go through a simple sign-up process. There are no age limits, so children from birth up to 18 years old can participate. Participants can expect to share their health information and complete some questionnaires over time, helping researchers understand how achondroplasia affects their lives and the impact of treatments like VOXZOGO™. This information will be valuable for improving care for children with this condition in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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
• Exclusion Criteria:
• • Lack of any medical records