NCT07301463

A Study in Children With Achondroplasia

A Multicenter, Longitudinal, Observational Study in Children With Achondroplasia

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 260 (estimated)
• Sponsor: Abbisko Therapeutics Co, Ltd · Industry
• Sex: All
• Age: 30 Months – 11 Years
• Healthy volunteers: Not accepted

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

Conditions

• Achondroplasia

Interventions

Type	Name	Description
OTHER	No Interventions	No Interventions
OTHER	complete a natural history observation of ACH for at least 6 months and up to 2 years	no interventions

Timeline

• Start date: 2025-06-20
• Primary completion: 2028-12-30
• Completion: 2039-04-30
• First posted: 2025-12-24
• Last updated: 2025-12-24

Locations

8 sites across 1 country: China