An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT ID: NCT03424018
Last Updated: 2025-01-09
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
119 participants
INTERVENTIONAL
2017-12-12
2031-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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BMN 111
BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Interventions
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BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
6 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Locations
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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
Countries
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References
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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.
Qi 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.
Related Links
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NIH Genetics Home Reference related topics: Achondroplasia
Description NIH Genetic and Rare Diseases Information Center resources: Achondroplasia
U.S. FDA Resources
Other Identifiers
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2017-002404-28
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
111-302
Identifier Type: -
Identifier Source: org_study_id
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