VIrtual STudy in Achondroplasia for the US (VISTA)

Recruiting now

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT06168201

Run by

BioMarin Pharmaceutical

People needed

170

Starts

2023-02-21

Expected to finish

2028-02-01

Last updated by the study team

2026-06-30

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Lack of any medical records

Where it is running

• UCSF — Oakland, California, United States (enrolling)
• PicnicHealth — San Francisco, California, United States (enrolling)
• Johns Hopkins University (JHU) — Baltimore, Maryland, United States (enrolling)
• Piedmont Health — Statesville, North Carolina, United States (enrolling)
• University of Cincinnati — Cincinnati, Ohio, United States (enrolling)
• Sanford Health — Sioux Falls, South Dakota, United States (enrolling)
• Baylor College of Medicine — Houston, Texas, United States (enrolling)
• University of Wisconsin — Madison, Wisconsin, United States (enrolling)

Full record on ClinicalTrials.gov

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