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

NCT06926491 | Recruiting Phase 3

• 2 other identifiers: 8398-001jRCT2031240562
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 9

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

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (1)

• Change from baseline in annualized height velocity

  Pre-dose, Week 52

Secondary Outcomes (1)

• Height Z-score using growth tables in patients with achondroplasia and the change from baseline

  Pre-dose, Week 52

Study Arms (1)

KK8398

EXPERIMENTAL

Drug: KK8398

Interventions

KK8398 DRUG

KK8398 will be administered.

KK8398

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• Patients who have been diagnosed with achondroplasia through genetic testing.

You may not qualify if:

• 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
• Patients who have received treatment with r-hGH or a CNP analogue within a certain period prior to the pre-enrollment examination.
• 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.

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (9)

Aichi Children's Health and Medical Center

Ōbu, Aichi-ken, Japan

RECRUITING

Osaka University Hospital

Suita, Osaka, Japan

RECRUITING

Osaka Women's and Children's Hospital

Waizumi, Osaka, Japan

RECRUITING

Tokyo Metropolitan Children's Medical Center

Fuchū, Tokyo, Japan

RECRUITING

Tottori University Hospital

Yonago, Tottori, Japan

RECRUITING

Niigata University Medical and Dental Hospital

Niigata, Japan

RECRUITING

Okayama University Hospital

Okayama, Japan

RECRUITING

ISEIKAI International General Hospital

Osaka, Japan

RECRUITING

Osaka City General Hospital

Osaka, Japan

RECRUITING

MeSH Terms

Conditions

Achondroplasia

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Yu Sato

  Kyowa Kirin Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Kyowa Kirin Co., Ltd.

CONTACT

+81-3-5205-7200; clinical.info.jp@kyowakirin.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 1, 2025
• First Posted: April 13, 2025
• Study Start: December 1, 2024
• Primary Completion (Estimated): May 31, 2029
• Study Completion (Estimated): May 31, 2031
• Last Updated: April 29, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

JP (9)