Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）

Phase 3

Recruiting

• Conditions: Achondroplasia
• Interventions: Drug: KK8398

• Registration Number: NCT06926491
• Lead Sponsor: Kyowa Kirin Co., Ltd.

Brief Summary

This trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia

Detailed Description

Not available

Study Timeline

• Study Start: 2024-12-01
• Status Verified: 2025-04
• Study First Submitted: 2025-04-01
• Study First Submitted QC: 2025-04-07
• Study First Posted: 2025-04-13
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-04-29
• Primary Completion: 2029-05-31
• Study Completion: 2031-05-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

Inclusion 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.

Exclusion 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 analogue 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
KK8398	KK8398	-

Primary Outcome Measures

Name	Time	Method
Change from baseline in annualized height velocity	Pre-dose, Week 52

Secondary Outcome Measures

Name	Time	Method
Height Z-score using growth tables in patients with achondroplasia and the change from baseline	Pre-dose, Week 52

Trial Locations

Locations (9)

Aichi Children's Health and Medical Center; 🇯🇵 Ōbu, Aichi, Japan

Osaka University Hospital; 🇯🇵 Suita, Osaka, Japan

Osaka Women's and Children's Hospital; 🇯🇵 Waizumi, Osaka, Japan

Tokyo Metropolitan Children's Medical Center; 🇯🇵 Fuchū, Tokyo, Japan

Tottori University Hospital; 🇯🇵 Yonago, Tottori, Japan

Niigata University Medical and Dental Hospital; 🇯🇵 Niigata, Japan

Okayama University Hospital; 🇯🇵 Okayama, Japan

ISEIKAI International General Hospital; 🇯🇵 Osaka, Japan

Osaka City General Hospital; 🇯🇵 Osaka, Japan