Growth drug shows promise for kids with dwarfism

NCT ID NCT03424018

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

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

Summary

This study tests a drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely helps them grow taller over several years. About 119 children who completed an earlier study will take the drug and be monitored for growth, daily activities, and quality of life.

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Conditions

The condition(s) this trial relates to.

achondroplasia Bone Diseases, Developmental bone disorder Dwarfism hereditary disease musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

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

ACHONDROPLASIA

Contacts and locations

Locations

Acibadem University School of Medicine

Istanbul, 34752, Turkey (Türkiye)
Alfred I. duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Ann and Robert H. Lurie Children's 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
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Emory University

Atlanta, Georgia, 30322, United States
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

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

Torrance, California, 90509, United States
Hospital Sant Joan de Deu

Barcelona, 08950, Spain
Hospital Universitario Virgen de la Victoria

Málaga, 29010, Spain
Institut Catala de Traumatologica I Medicina de l'Esport

Barcelona, 08028, Spain
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, 53226, United States
Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia
Osaka University Hospital

Osaka, Japan
Otto-von-Gericke Universitaet, Universitaetskinderklinik

Magdeburg, 39120, Germany
Saitama Children's Medical Center

Saitama, Japan
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Sheffield Children's NHS Foundation Trust

Sheffield, S10 2TH, United Kingdom
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
Tokushima University Hospital

Tokushima, Japan
University of Missouri

Columbia, Missouri, 65201, United States
Universitätsklinikum Münster

Münster, 48149, Germany

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