An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Running, not enrolling · Phase 3

Conditions studied: Achondroplasia

In brief

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Key facts

Study ID

NCT03424018

Run by

BioMarin Pharmaceutical

People needed

119

Starts

2017-12-12

Expected to finish

2031-06-01

Last updated by the study team

2026-03-13

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Must have completed Study 111-301
• Female >= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study
• If sexually active, willing to use a highly effective method of contraception while participating in the study
• Are willing and able to perform all study procedures
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.

You may not qualify if…

• Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study
• Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function
• Evidence of decreased growth velocity (<1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays.
• Require any investigational agent prior to completion of study period
• Current therapy with medications known to alter renal function
• Pregnant or breastfeeding or plan to become pregnant during study
• Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
• 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.

Where it is running

• Children's Hospital & Research Center Oakland — Oakland, California, United States
• Harbor - UCLA Medical Center — Torrance, California, United States
• Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• Emory University — Atlanta, Georgia, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• University of Missouri — Columbia, Missouri, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Baylor College of Medicine — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin, United States
• The Children's Hospital at Westmead — Westmead, New South Wales, Australia
• Murdoch Children's Research Institute — Parkville, Victoria, Australia
• Otto-von-Gericke Universitaet, Universitaetskinderklinik — Magdeburg, Germany
• Universitätsklinikum Münster — Münster, Germany
• Osaka University Hospital — Osaka, Japan
• Saitama Children's Medical Center — Saitama, Japan
• Tokushima University Hospital — Tokushima, Japan
• Institut Catala de Traumatologica I Medicina de l'Esport — Barcelona, Spain
• Hospital Sant Joan de Deu — Barcelona, Spain
• Hospital Universitario Virgen de la Victoria — Málaga, Spain
• Acibadem University School of Medicine — Istanbul, Turkey (Türkiye)
• Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital — London, United Kingdom
• Sheffield Children's NHS Foundation Trust — Sheffield, United Kingdom

Full record on ClinicalTrials.gov

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