A Study in Children With Achondroplasia
brief summary
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural history observation of ACH for at least 6 months and up to 2 years.
detailed description
Primary Objectives To evaluate the safety, tolerability, and recommended dose for expansion (RDE) of oral ABSK061 in children with ACH To evaluate the efficacy of oral ABSK061 in children with ACH
Secondary Objectives To characterize the pharmacokinetics (PK) of ABSK061 and potential disproportional metabolites (if applicable) To evaluate changes from baseline in anthropometric parameters after administration of oral ABSK061 To evaluate the acceptability of ABSK061 minitablets for peroral administration in children with ACH
Exploratory Objectives To evaluate changes in ACH complications and disease burden after oral administration of ABSK061 To evaluate the pharmacodynamic (PD) profile in children with ACH after oral administration of ABSK061
official title
A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia