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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
119
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Change from baselines in mean annualized growth velocity
Long term efficacy as measured by change in annualized growth velocity
Time frame: Through study completion, an average of 1 year
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
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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
...and 14 more locations
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
BMN 111 activity will be assessed by measuring bone and collagen metabolism
Time frame: Through study completion, every 12 months
Evaluate change from baseline in body proportion ratios of the extremities
Time frame: Through study completion, every 6 months
Effect of BMN 111 on bone morphology and quality
The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via X-Ray and Dual X-ray Absorptiometry
Time frame: Through study completion, every 12 months for DXA or 2 years for X-ray
Final Adult Height
Height at 16 years for females and 18 years for males
Time frame: Up to at least 16 years of age for females and 18 years of age for males