NCT06433557

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

A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Ascendis Pharma Growth Disorders A/S · Industry
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections	For navepegritide, a once weekly s.c. dose of 100 μg CNP/kg. For lonapegsomatropin, a once weekly s.c. dose of lonapegsomatropin 0.30 mg hGH/kg as starting dose. Treatment duration of up to 156 weeks.

Timeline

• Start date: 2024-07-26
• Primary completion: 2025-11-14
• Completion: 2027-11-01
• First posted: 2024-05-29
• Last updated: 2026-02-18

Locations

3 sites across 3 countries: Denmark, Ireland, United Kingdom

Regulatory

• FDA-regulated drug study