DRUG
BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Status
Active, not recruiting
Phase
Phase 3
Enrollment
119
Locations
24
Results
Not posted
Publications
3
Study summary
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
Interventions
DRUG
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Timeline
First posted
Feb 6, 2018
Study start
Dec 12, 2017
Primary completion
Jun 2031
Study completion
Jun 2031
Results posted
Not reported
Registry updated
Mar 13, 2026
Outcomes
Change from baselines in mean annualized growth velocity
Time frame · Through study completion, an average of 1 year
Long term efficacy as measured by change in annualized growth velocity
Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire
Time frame · Through study completion, every 6-12 months
Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire
Time frame · Through study completion, every 12 months
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Time frame · Through study completion, every 12 months
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame · Through study completion, every 12 months
Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame · Through study completion, every 12 months
Characterize the elimination half-life of BMN 111 (t1⁄2)
Time frame · Through study completion, every 12 months
Characterize the apparent clearance of drug
Time frame · Through study completion, every 12 months
Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)
Time frame · Through study completion, every 12 months
Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)
Time frame · Through study completion, every 12 months
BMN 111 Activity Biomarkers
Time frame · Through study completion, every 12 months
BMN 111 activity will be assessed by measuring bone and collagen metabolism
Eligibility
Inclusion Criteria: * 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. Exclusion Criteria: * 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.
Study locations
Australia · Germany · Japan · Spain · Turkey (Türkiye) · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
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
Publications
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