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

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.

Timeline

Start

2023-03-03

Primary completion

2024-08-09

Completion

2025-08-13

Outcome

Met primary endpoint

registry analysis (superiority test); Navepegritide vs Placebo for Navepegritide; p <0.0001; Least Square Mean Difference 1.49 (95% CI 1.05 to 1.93); ANCOVA NCT05598320 ↗

Publications

• McDonnell CM, Irving M, Nolting LA, Abdelrahman SG, Dalby LW, Ikle JM, Jensen SM, Komirenko AS, Ominsky MS, Shu AD, Hove HB. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026 Jun 1;194(6):745-755. doi: 10.1093/ejendo/lvag082.
• Savarirayan R, McDonnell C, Bacino CA, Hoernschemeyer DG, Legare JM, Abuzzahab MJ, Hofman PL, Campeau PM, de Bergua Domingo JM, Ward LM, Smit K, Smith A, Mao M, Ominsky MS, Freiberg LC, Shu AD, Hove HB. Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr. 2026 Jan 1;180(1):18-25. doi: 10.1001/jamapediatrics.2025.4771.

Drugs

Indications