The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period was followed by an Open Label Extension (OLE) period of a 52-week duration.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
84
Once-weekly subcutaneous injection of 100 µg/kg Navepegritide (TransCon CNP)
Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide (TransCon CNP)
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, United States
Ascendis Pharma Investigational Site
Columbia, Missouri, United States
Ascendis Pharma Investigational Site
Houston, Texas, United States
Annualized Growth Velocity (AGV) at Week 52
Annualized growth velocity is defined as (height - baseline height)/(date of height assessment - date of baseline) \* 365.25. Annualized growth velocity reported in terms of centimeters (cm) per year. Missing values at Week 52 were imputed by a multiple imputation method.
Time frame: At Week 52
Change From Baseline in Height Z-score (ACH-specific) at Week 52
ACH specific Z-scores of height provide a measure of growth relative to other individuals with ACH from the CLARITY database. A height Z-score is a standardized height measure after considering important factors like age and gender. Z-scores (or standard deviation scores) describe how far the measurement deviates from the mean. A height Z-score of 0 indicates that height is equal to the mean in the reference population. Negative numbers indicate values below the mean and positive numbers indicate values above the mean.
Time frame: Baseline, Week 52
Change From Baseline in Height Z-score (CDC-Based) at Week 52
Z-scores of height are determined using Centers for Disease Control and Prevention (CDC) clinical growth charts for children. A height Z-score is a standardized height measure after considering important factors like age and gender. Z-scores (or standard deviation scores) describe how far the measurement deviates from the median. A height Z-score of 0 indicates that height is equal to the median in the reference population. Negative numbers indicate values below the median and positive numbers indicate values above the median.
Time frame: Baseline, Week 52
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Ascendis Pharma Investigational Site
Madison, Wisconsin, United States
Ascendis Pharma Investigational Site
Parkville, Australia
Ascendis Pharma Investigational Site
Montreal, Canada
Ascendis Pharma Investigational Site
Copenhagen, Denmark
Ascendis Pharma Investigational Site
Dublin, Ireland
Ascendis Pharma Investigational Site
Auckland, New Zealand
Ascendis Pharma Investigational Site
Vitoria-Gasteiz, Spain