Phase 2 Study of Weekly Subcutaneous TransCon CNP Treatment for Infants Under 2 Years with Genetically Confirmed Achondroplasia

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide TransCon CNP in infants with genetically confirmed achondroplasia ACH who are younger than 2 years old. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial aims to compare weekly doses of Navepegritide with a placebo over a 52-week period to understand its impact on growth and safety in this population. Participants receive once-weekly subcutaneous injections of either 100 bcgkg Navepegritide or a placebo for 52 weeks. The study is randomized in a 21 ratio, with some infants receiving the active drug and others receiving placebo injections. After the 52-week treatment period, there is an open-label extension phase allowing continued evaluation. During the study, infants will be monitored through medical history reviews, physical exams, vital signs, ECGs, imaging, and lab tests to evaluate safety and growth changes. The main outcomes measured are the safety and tolerability of Navepegritide and its effect on growth over 52 weeks. Parents or caregivers will administer weekly injections and follow study instructions, including vitamin D supplementation where applicable. The total participation time includes the initial 52 weeks of treatment followed by further observation during the extension period.

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