NIPH Clinical Trials Search

Phase 3 study to evaluate the efficacy and safety of KK8398 in patients with achondroplasia (AOBA study)

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Achondroplasia
Date of first enrollment	25/04/2025
Target sample size	6
Countries of recruitment
Study type	Interventional
Intervention(s)	After obtaining written consent, patients with achondroplasia will have their previous treatment(s) washed out if necessary, and then begin treatment with KK8398.

Outcome(s)

Primary Outcome	Change from Baseline in annualized height velocity
Secondary Outcome	Change from Baseline at each evaluation point in annualized height velocity

Key inclusion & exclusion criteria

Age minimum	>= 3age old
Age maximum	< 18age old
Gender	Both
Include criteria	1) Patients who are between 2.5 and 17.5 years old of age at the time of provisional registration. Patients who meet certain criteria in terms of Tanner stage and bone age. 2) Patients who have been diagnosed with achondroplasia through genetic testing.
Exclude criteria	1) Patients who meet the criteria for a certain height determined by age and gender in the "Growth Chart for Patients with Achondroplasia" at the time of the pre-registration examination. 2) Patients who have received treatment with r-hGH or a CNP analogat within a certain period prior to the pre-enrollment examination. 3) Patients who have had previous osteotomy or who are scheduled to undergo osteotomy or epiphyseal growth inhibition during the study period. Epiphyseal growth inhibition in which the plate was removed more than 26 weeks prior to the pre-registration examination will not be excluded if the patient has completely healed without sequelae, based on the judgment of the investigator or sub-investigator.