TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
140
TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.
Ascendis Pharma Investigational Site
Little Rock, Arkansas, United States
Ascendis Investigational Site
Aurora, Colorado, United States
Ascendis Investigational Site
Wilmington, Delaware, United States
Ascendis Investigational Site
Saint Paul, Minnesota, United States
Ascendis Pharma Investigational Site
Columbia Falls, Montana, United States
Ascendis Pharma Investigational Site
Buffalo, New York, United States
Ascendis Investigational Site
Houston, Texas, United States
Ascendis Pharma Investigational Site
Madison, Wisconsin, United States
Ascendis Pharma Investigational Site
Parkville, Victoria, Australia
Ascendis Investigational Site
Linz, Austria
...and 7 more locations
Safety and Tolerability
Incidence of Treatment-Emergent Adverse Events
Time frame: Through trial completion, an average of 10 years
Height Z-scores
Number of standard deviations
Time frame: Through trial completion, an average of 10 years
Annualized Growth Velocity
cm per year
Time frame: Through trial completion, an average of 10 years
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