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A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
Sponsor: Ascendis Pharma Growth Disorders A/S
Summary
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.
Official title: A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children and Adolescents With Achondroplasia
Key Details
Gender
All
Age Range
3 Years - 15 Years
Study Type
INTERVENTIONAL
Enrollment
140
Start Date
2023-06-21
Completion Date
2039-03
Last Updated
2025-10-27
Healthy Volunteers
No
Conditions
Interventions
TransCon CNP
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.
Locations (17)
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
Ascendis Investigational Site
Montreal, Canada
Ascendis Pharma Investigational Site
Copenhagen, Denmark
Ascendis Investigational Site
Berlin, Germany
Ascendis Pharma Investigational Site
Dublin, Ireland
Ascendis Investigational Site
Auckland, New Zealand
Ascendis Investigational Site
Coimbra, Portugal
Ascendis Investigational Site
Vitoria-Gasteiz, Spain