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An Open-Label, Uncontrolled, Single Arm Clinical Trial of ACP-015 in Children with Achondroplasia Aged 2 to 11 Years.

Basic Information

Recruitment status	Not Recruiting
Health condition(s) or Problem(s) studied	Achondroplasia
Date of first enrollment	19/11/2025
Target sample size	12
Countries of recruitment
Study type	Interventional
Intervention(s)	Navepegritide will be administered subcutaneously once weekly at a dose of 100 microg/kg. The duration of treatment will be 52 weeks.

Outcome(s)

Primary Outcome	Change in ACH-specific height Z-score (Z-score using the growth curve of untreated ACH patients as the reference) from baseline to Week 52
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 2age old
Age maximum	<= 11age old
Gender	Both
Include criteria	-Male or female, between 2 and 11 years of age (inclusive) at the time of informed consent. -Have been genetically confirmed as ACH and have a clinical diagnosis at the time of screening test (documentation of past test results is available as proof of diagnosis). -Able to stand without assistance. -Parent(s) or legal guardian(s) are willing to comply with the protocol and able to administer once-weekly subcutaneous injections of investigational product. -Treatment with vosoritide at the time of screening test which is continued for the last >=26 weeks (for patients previously treated with somatropin, at least 12 months must have passed between the last dose of somatropin and the time of screening test).
Exclude criteria	-Participation in an interventional clinical study within 3 months prior to screening test. -Radiographically confirmed epiphyseal closure or a low annualized growth velocity (AGV <1.5 cm/year) at screening test for navepegritide. -Have a growth disorder other than ACH that results in short stature or abnormal growth. 1)Severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN). 2)Hypochondroplasia, pseudoachondroplasia, or any skeletal system disorder similar to ACH. 3)Growth hormone deficiency, Turner syndrome. -Have any of the following past history or complications that may affect growth: 1)Inflammatory bowel disease. 2)Celiac disease. 3)Hypothyroidism and/or hyperthyroidism. 4)Pre-diabetes or diabetes mellitus. -Have a history of the following cardiac abnormalities or clinically significant findings at screening test (but not limited to the following): 1)Repaired or unrepaired coarctation. 2)Moderate or greater congenital heart disease including tetralogy of Fallot, atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease. 3)QTcF >=450 msec on 12-lead electrocardiogram. -Known history or presence of malignant disease.