Long-Term Observational Study of Oral ABSK061 in Children Aged 2.5 to Under 11 Years with Achondroplasia

What this Trial Is About

Researchers are conducting an observational study to gather detailed information about children with achondroplasia ACH, a form of dwarfism. The study aims to collect data on body measurements, clinical features, related medical conditions, quality of life, and treatments over a period ranging from 6 months to 2 years. The study also evaluates the safety, tolerability, and appropriate dosing of an oral medication called ABSK061, as well as its effects on growth and disease burden in children with ACH. Participants aged 2.5 to under 11 years old, who have a confirmed diagnosis of ACH through genetic testing, will be observed without any interventions. The study will monitor changes in growth parameters such as annualized growth velocity, standing height, sitting height, and their ratios. Additionally, researchers will assess how well children accept the ABSK061 minitablets taken orally. The study includes safety evaluations and pharmacokinetic and pharmacodynamic assessments of the medication. During the study, childrens growth and health will be regularly measured every three months for up to two years. Assessments include physical measurements, clinical evaluations, and monitoring for ACH complications. Researchers will observe how the disease progresses naturally and how the oral medication ABSK061 influences growth and disease-related factors. The study ensures ongoing safety monitoring and allows participants to be ambulatory and capable of standing without assistance throughout the observation period.

A Study in Children With Achondroplasia