VIrtual STudy in Achondroplasia for the US (VISTA) (NCT06168201)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UCSF — Oakland, California
Age range: 0 Months to 18 Years
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (8 Sites)
- UCSF — Oakland, California
- PicnicHealth — San Francisco, California
- Johns Hopkins University (JHU) — Baltimore, Maryland
- Piedmont Health — Statesville, North Carolina
- University of Cincinnati — Cincinnati, Ohio
- Sanford Health — Sioux Falls, South Dakota
- Baylor College of Medicine — Houston, Texas
- University of Wisconsin — Madison, Wisconsin
📞 Contact the Study Coordinator
Phone: (800) 938-4587
Email: medinfo@bmrn.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Achondroplasia
- A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) — Oakland, California
- An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia — Oakland, California
- An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia — Oakland, California
- Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) — Oakland, California
- A Study of TYRA-300 in Children With Achondroplasia: BEACH301 — Torrance, California
Explore by condition: All Achondroplasia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.