This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
BMN 111 will be administered subcutaneously daily.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Harbor - UCLA Medical Center
Torrance, California, United States
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Growth Velocity [Efficacy]
Annualized growth velocity (cm/yr)
Time frame: Until near final adult height is reached
Growth Parameters (Efficacy)
Height standard score (Z-score)
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Body Proportions (Efficacy)
Upper-to-lower body segment ratio
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Final Adult Height
Height at the age of 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
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Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, United States
Vanderbilt University
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Institut Necker
Paris, France
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, United Kingdom