6 recruiting studies matching your filters
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with …
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participant…
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with AC…
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated …
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety,…
The purpose of this study is to create an electronic registry to house phenotypic information from patients with achondroplasia. The initial focus of this regi…