A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia

Phase 2 Active not recruiting 22 enrolled Ascendis Pharma Growth Disorders A/S

NaSingle-groupOpen-labelTreatment

Summary

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

Timeline

Start: 2024-07-26
Primary completion: 2025-11-14
Completion: 2027-11

Outcome

Met primary endpoint

paper least squares mean AGV of 8.69 cm/year versus 5.95 cm/year for matched children who received navepegritide monotherapy, a difference of 2.74 cm/year (P < .0001). PMID 42144862 ↗

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Lonapegsomatropin	Protein / enzyme biologic	0.3 mg/kg	Subcutaneous
Subject	Navepegritide	Protein / enzyme biologic	100 ug/kg	Subcutaneous