drugset / Trial / NCT04085523
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
Timeline
- Start
- 2020-06-24
- Primary completion
- 2022-09-27
- Completion
- 2024-10-01
Publications
- Results Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zarate YA, Bacino CA, Bober MB, Legare JM, Hogler W, Quattrin T, Abuzzahab MJ, Hofman PL, White KK, Ma NS, Schnabel D, Sousa SB, Mao M, Smith A, Chakraborty M, Giwa A, Winding B, Volck B, Shu AD, McDonnell C. Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial. EClinicalMedicine. 2023 Oct 2;65:102258. doi: 10.1016/j.eclinm.2023.102258. eCollection 2023 Nov.
- Brod M, McLeod L, Smith A. Assessing signs and impacts of achondroplasia: psychometric evaluation of the Achondroplasia Child Experience Measures. J Patient Rep Outcomes. 2026 Jun 19. doi: 10.1186/s41687-026-01123-z. Online ahead of print.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Navepegritide | Protein / enzyme biologic | 6 ug/kg | Subcutaneous |
| Subject | Navepegritide | Protein / enzyme biologic | 20 ug/kg | Subcutaneous |
| Subject | Navepegritide | Protein / enzyme biologic | 50 ug/kg | Subcutaneous |
| Subject | Navepegritide | Protein / enzyme biologic | 100 ug/kg | Subcutaneous |