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Clinical Trials in the USA / NCT02724228
Active, not recruiting Phase 2

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

NCT02724228 · tracked via the Priya Life Science USA tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2016-01-26
Last updated
2026-03-13

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

BMN 111

BMN 111: BMN 111 will be administered subcutaneously daily.

Study summary

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.

Eligibility

Sex
ALL
Min age
7 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * 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. 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.

Primary outcome measure(s)

Trial sites (9)

FacilityCityRegionStatus
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 France
Guys & St. Thomas NHS Foundation Trust Evelina Hospital London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02724228 on ClinicalTrials.gov ↗ ← All trials in the USA