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Clinical Trials in the UK / NCT03424018
Active, not recruiting Phase 3

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

NCT03424018 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 3
Started
2017-12-12
Last updated
2026-03-13

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

BMN 111

BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

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

Eligibility

Sex
ALL
Min age
6 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (24)

FacilityCityRegionStatus
Children's Hospital & Research Center Oakland Oakland California
Harbor - UCLA Medical Center Torrance California
Alfred I. duPont Hospital for Children Wilmington Delaware
Emory University Atlanta Georgia
Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
Johns Hopkins University Baltimore Maryland
University of Missouri Columbia Missouri
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
Baylor College of Medicine Houston Texas
Seattle Children's Hospital Seattle Washington
Medical College of Wisconsin, Children's Hospital Milwaukee Wisconsin
The Children's Hospital at Westmead Westmead New South Wales
Murdoch Children's Research Institute Parkville Victoria
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
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 NCT03424018 on ClinicalTrials.gov ↗ ← All trials in the UK