Hope for growth: new drug shows promise in Long-Term achondroplasia study

NCT ID NCT02724228

Ongoing Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety and effects of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. It includes 30 children who have already taken the drug for two years in a previous study. The goal is to see if the drug remains safe and can help improve growth over time.

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Conditions

The condition(s) this trial relates to.

achondroplasia Bone Diseases, Developmental bone disorder Dwarfism

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACHONDROPLASIA

Contacts and locations

Locations

Ann and Robert H. Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States
Baylor College of Medicine

Houston, Texas, 77030, United States
Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States
Guys & St. Thomas NHS Foundation Trust Evelina Hospital

London, SE1 9RT, United Kingdom
Harbor - UCLA Medical Center

Torrance, California, 90509, United States
Institut Necker

Paris, 75015, France
Johns Hopkins McKusick - Institute of Genetic Medicine

Baltimore, Maryland, 21287, United States
Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia
Vanderbilt University

Nashville, Tennessee, 37232-2578, United States

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Other studies related to the condition(s) this trial covers.