NCT05598320

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

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: Ascendis Pharma Growth Disorders A/S · Industry
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly SC doses of 100 µg 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 is followed by an Open Label Extension (OLE) period of a 52-week duration.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	TransCon CNP	Once-weekly subcutaneous injection of 100 µg/kg TransCon CNP
DRUG	Placebo for TransCon CNP	Once-weekly subcutaneous injection of 100 µg/kg placebo for TransCon CNP

Timeline

• Start date: 2023-03-03
• Primary completion: 2024-08-09
• Completion: 2025-08-13
• First posted: 2022-10-28
• Last updated: 2026-01-15

Locations

10 sites across 7 countries: United States, Australia, Canada, Denmark, Ireland, New Zealand, Spain

Regulatory

• FDA-regulated drug study