NCT06079398

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 72 (estimated)
• Sponsor: Ascendis Pharma A/S · Industry
• Sex: All
• Age: 0 Years – 2 Years
• Healthy volunteers: Not accepted

Summary

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.

Conditions

• Achondroplasia

Interventions

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

Timeline

• Start date: 2024-01-23
• Primary completion: 2027-12-01
• Completion: 2028-12-01
• First posted: 2023-10-12
• Last updated: 2026-04-08

Locations

18 sites across 15 countries: United States, Australia, Austria, Canada, Denmark, Finland, France, Germany, Ireland, Italy, New Zealand, Norway, Portugal, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study