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.
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
Study Type
OBSERVATIONAL
Enrollment
260
No Interventions
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGGuangzhou Women and Childrens Medical Center
Guangzhou, Guangzhou, China
NOT_YET_RECRUITINGAnnualized growth velocity (AGV)
Annualized growth velocity (AGV)
Time frame: Through the study completion, an average of three months, up to 2 years
standing height
calculated to the nearest 0.1 cm
Time frame: an average of three months, up to 2 years
sitting height
calculated to the nearest 0.1 cm
Time frame: an average of three months, up to 2 years
sitting height to standing height ratio
This parameter is calculated as the ratio of sitting height to total standing height. It is used to assess the abnormality in body proportions, specifically the relative trunk-to-lower limb length. In patients with achondroplasia, this ratio is typically increased. Within the clinical trial, this measure is used to evaluate the treatment drug's effect on body proportions.
Time frame: an average of three months, up to 2 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Henan Children's Hospital, Zhengzhou Children's Hospital
Zhengzhou, Henan, China
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
RECRUITINGXin Hua Hospital Affiliatod to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
RECRUITINGChengdu Women's and Children's Central Hospital
Chengdu, Sichuan, China
NOT_YET_RECRUITINGWest China Second University Hospital, Sichuan University
Chengdu, Sichuan, China
ACTIVE_NOT_RECRUITINGChildren's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
NOT_YET_RECRUITING