An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia (NCT03989947)
A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
🩺 Plain-English Summary (8th-Grade Level)
This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital & Research Center Oakland — Oakland, California
Age range: 15 Months
🧪 Interventions in This Study
📋 "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 (16 Sites)
- Children's Hospital & Research Center Oakland — Oakland, California
- Harbor - UCLA Medical Center — Torrance, California
- Alfred I. duPont Hospital for Children — Wilmington, Delaware
- Emory University — Decatur, Georgia
- Ann Robert and H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- Cincinnati Childrens Hospital — Cincinnati, Ohio
- Vanderbilt University Medical Center — Nashville, Tennessee
- Baylor College of Medicine — Houston, Texas
- Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin
- The Children's Hospital at Westmead — Westmead, New South Wales
- Murdoch Children's Research Institute — Parkville, Victoria
- Osaka University — Osaka,
- Saitama Children's Medical Center — Saitama,
- Tokushima University Hospital — Tokushima,
- Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital — London,
- Sheffield Children's NHS Foundation Trust — Sheffield,
💰 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 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
- VIrtual STudy in Achondroplasia for the US (VISTA) — Oakland, California
Explore by condition: All Achondroplasia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.