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ENROLLING BY INVITATION
NCT05929807
PHASE2/PHASE3

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

View on ClinicalTrials.gov

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

DRUG

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