๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-03-12

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) (NCT02724228)

A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia

๐Ÿ”ต Active, Not Recruiting Phase2 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: BioMarin Pharmaceutical

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

Who can join: Review the eligibility criteria below.

Location: Children's Hospital & Research Center Oakland โ€” Oakland, California

Age range: 7 Years

๐Ÿงช Interventions in This Study

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (9 Sites)

  • Children's Hospital & Research Center Oakland โ€” Oakland, California
  • Harbor - UCLA Medical Center โ€” Torrance, California
  • Ann and Robert H. Lurie Childrens Hospital of Chicago โ€” Chicago, Illinois
  • Johns Hopkins McKusick - Institute of Genetic Medicine โ€” Baltimore, Maryland
  • Vanderbilt University โ€” Nashville, Tennessee
  • Baylor College of Medicine โ€” Houston, Texas
  • Murdoch Children's Research Institute โ€” Parkville, Victoria
  • Institut Necker โ€” Paris,
  • Guys & St. Thomas NHS Foundation Trust Evelina Hospital โ€” London,

๐Ÿ’ฐ 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 ยง 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.

Explore by condition: All Achondroplasia clinical trials

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—