Observational study of achondroplasia in the US
Official title: VIrtual STudy in Achondroplasia for the US (VISTA)
Run by BioMarin Pharmaceutical · NCT06168201 · Data updated 2026-11-12
This study looks at children in the U.S. with achondroplasia to see how they are doing with and without the treatment VOXZOGO™ over at least five years.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 0 Months to 18 Years |
| Sex | All |
| Enrollment | 170 (estimated) |
| Sponsor | BioMarin Pharmaceutical Industry-sponsored |
| Locations | 8 sites (Oakland, California and 7 other locations) |
| Status | Recruiting |
| Started | February 21, 2023 |
| Main results expected | February 2028 |
| Study ends | February 2028 |
| Registry record updated | June 30, 2026 |
| Trial ID | NCT06168201 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals diagnosed with achondroplasia, aged 13 years or younger, receiving medical care in the United States.
✕Probably not a fit
Patients without any medical records will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study focuses on individuals with achondroplasia in the United States, particularly pediatric patients treated and untreated with VOXZOGO™. It aims to collect data over a minimum of five years through participant-mediated access to electronic health records and clinical outcome assessments. The study will gather both retrospective and prospective data to reflect real-life treatment use and clinical care. Enrollment began in February 2023, and the study may be extended based on sponsor decisions.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the real-world effectiveness and safety of treatments for achondroplasia.
How similar studies have gone: While this study is observational, similar studies have shown success in understanding treatment impacts in rare conditions, making this approach promising.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
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UCSF
Oakland, California, United States Recruiting -
PicnicHealth
San Francisco, California, United States Recruiting -
Johns Hopkins University (JHU)
Baltimore, Maryland, United States Recruiting -
Piedmont Health
Statesville, North Carolina, United States Recruiting -
University of Cincinnati
Cincinnati, Ohio, United States Recruiting -
Sanford Health
Sioux Falls, South Dakota, United States Recruiting
Show 2 more locations
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Baylor College of Medicine
Houston, Texas, United States Recruiting -
University of Wisconsin
Madison, Wisconsin, United States Recruiting
Study contacts
- Study coordinator: Trial Specialist
- Email: medinfo@bmrn.com
- Phone: 1-800-938-4587
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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BMN 333 versus daily vosoritide for growth in children with achondroplasia
This trial tests whether weekly BMN 333 injections help children with achondroplasia grow as well as daily vosoritide.
-
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This project will try using Syde® wearable movement recordings to see which measurements reliably track limb movement in people aged 3 and older with achondroplasia or hypochondroplasia.
-
Observing children with achondroplasia over time
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Infigratinib treatment for children under 3 with achondroplasia
This trial tests whether daily oral infigratinib helps children younger than 3 years who have achondroplasia.
-
Evaluating TYRA-300 for children with achondroplasia
This study is testing a new medication called TYRA-300 to see if it is safe and helps children aged 3 to 10 with achondroplasia grow better.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.