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COMPLETED
NCT05598320
PHASE2/PHASE3

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

Sponsor: Ascendis Pharma Growth Disorders A/S

View on ClinicalTrials.gov

Summary

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.

Official title: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children With Achondroplasia Followed by an Open Label Extension Period

Key Details

Gender

All

Age Range

2 Years - 11 Years

Study Type

INTERVENTIONAL

Enrollment

84

Start Date

2023-03-03

Completion Date

2025-08-13

Last Updated

2026-07-14

Healthy Volunteers

No

Conditions

Interventions

DRUG

Navepegritide (TransCon CNP)

Once-weekly subcutaneous injection of 100 µg/kg Navepegritide (TransCon CNP)

DRUG

Placebo for Navepegritide (TransCon CNP)

Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide (TransCon CNP)

Locations (10)

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

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