Observational study of achondroplasia in the US
VIrtual STudy in Achondroplasia for the US (VISTA)
BioMarin Pharmaceutical · NCT06168201
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 |
|---|---|
| Enrollment | 170 (estimated) |
| Ages | 0 Months to 18 Years |
| Sex | All |
| Sponsor | BioMarin Pharmaceutical (industry) |
| Locations | 7 sites (Oakland, California and 6 other locations) |
| Trial ID | NCT06168201 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with achondroplasia, aged 13 years or younger, receiving medical care in the United States.
Not a fit: Patients without any medical records will not benefit from this study.
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 performed: While this study is observational, similar studies have shown success in understanding treatment impacts in rare conditions, making this approach promising.
Eligibility criteria
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 this trial is running
Oakland, California and 6 other locations
- UCSF — Oakland, California, United States (RECRUITING)
- PicnicHealth — San Francisco, California, 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)
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Achondroplasia