An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
2 other identifiers
interventional
119
7 countries
24
Brief 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2017
Longer than P75 for phase_3
24 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 12, 2017
CompletedFirst Submitted
Initial submission to the registry
December 28, 2017
CompletedFirst Posted
Study publicly available on registry
February 6, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2031
March 13, 2026
March 1, 2026
13.5 years
December 28, 2017
March 12, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from baselines in mean annualized growth velocity
Long term efficacy as measured by change in annualized growth velocity
Through study completion, an average of 1 year
Secondary Outcomes (13)
Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire
Through study completion, every 6-12 months
Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire
Through study completion, every 12 months
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Through study completion, every 12 months
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)
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)
Through study completion, every 12 months
- +8 more secondary outcomes
Other Outcomes (1)
Optional exploratory genomic biomarker analysis
Once through study completion
Study Arms (1)
BMN 111
EXPERIMENTALInterventions
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (24)
Children's Hospital & Research Center Oakland
Oakland, California, 94609, United States
Harbor - UCLA Medical Center
Torrance, California, 90509, United States
Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803, United States
Emory University
Atlanta, Georgia, 30322, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
University of Missouri
Columbia, Missouri, 65201, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, 53226, United States
The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
Otto-von-Gericke Universitaet, Universitaetskinderklinik
Magdeburg, 39120, Germany
Universitätsklinikum Münster
Münster, 48149, 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, 08028, Spain
Hospital Sant Joan de Deu
Barcelona, 08950, Spain
Hospital Universitario Virgen de la Victoria
Málaga, 29010, Spain
Acibadem University School of Medicine
Istanbul, 34752, Turkey (Türkiye)
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, SE1 9RT, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH, United Kingdom
Related Publications (2)
Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025 May 9;6(5):100566. doi: 10.1016/j.medj.2024.11.019. Epub 2024 Dec 30.
PMID: 39740666DERIVEDQi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23.
PMID: 38649657DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 28, 2017
First Posted
February 6, 2018
Study Start
December 12, 2017
Primary Completion (Estimated)
June 1, 2031
Study Completion (Estimated)
June 1, 2031
Last Updated
March 13, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share