Who is eligible for clinical trial NCT02724228?

Participants must meet protocol criteria for Achondroplasia. Confirm exact inclusion and exclusion requirements with the study coordinator.

Does clinical trial NCT02724228 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-03-12

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

🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic Sponsor: BioMarin Pharmaceutical

🩺 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: To join this study, participants generally need to meet the listed inclusion criteria (6 items) and avoid the listed exclusions (7 items).

Location: Children's Hospital & Research Center Oakland — Oakland, California

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Have completed 24 months of BMN 111 treatment in Study 111-202.
Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
If sexually active, willing to use a highly effective method of contraception while participating in the study.
Females \>= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
Willing and able to perform all study procedures as physically possible
Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

Usually NOT eligible if any of the following apply:

Exclusion Criteria:
Requires any investigational agent prior to completion of study period.
Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
Permanently discontinued BMN 111 during the 111-202 study.
Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
Current chronic therapy with restricted medications.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (9 Sites)

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

💰 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.

🏛️ View Official NIH Record ↗