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

April 28, 2025 updated by: Kyowa Kirin Co., Ltd.

A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

6

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Japan
      • Niigata, Japan
        • Recruiting
        • Niigata University Medical And Dental Hospital
      • Okayama, Japan
        • Recruiting
        • Okayama University Hospital
      • Osaka, Japan
        • Recruiting
        • Osaka City General Hospital
      • Osaka, Japan
        • Recruiting
        • Iseikai International General Hospital
    • Aichi
      • Ōbu, Aichi, Japan
        • Recruiting
        • Aichi Children's Health and Medical Center
    • Osaka
      • Suita, Osaka, Japan
        • Recruiting
        • Osaka University Hospital
      • Waizumi, Osaka, Japan
        • Recruiting
        • Osaka Women's and Children's Hospital
    • Tokyo
      • Fuchū, Tokyo, Japan
        • Recruiting
        • Tokyo Metropolitan Children's Medical Center
    • Tottori
      • Yonago, Tottori, Japan
        • Recruiting
        • Tottori University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: KK8398
Drug: KK8398
KK8398 will be administered.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in annualized height velocity
Time Frame: Pre-dose, Week 52
Pre-dose, Week 52

Secondary Outcome Measures

Outcome Measure
Time Frame
Height Z-score using growth tables in patients with achondroplasia and the change from baseline
Time Frame: Pre-dose, Week 52
Pre-dose, Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kyowa Kirin Co., Ltd.

Investigators

  • Study Director: Yu Sato, Kyowa Kirin Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2024

Primary Completion (Estimated)

May 31, 2029

Study Completion (Estimated)

May 31, 2031

Study Registration Dates

First Submitted

April 1, 2025

First Submitted That Met QC Criteria

April 7, 2025

First Posted (Actual)

April 13, 2025

Study Record Updates

Last Update Posted (Actual)

April 29, 2025

Last Update Submitted That Met QC Criteria

April 28, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 8398-001
  • jRCT2031240562 (Registry Identifier: Japan Registry of Clinical Trials (jRCT))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

There is not a plan to make IPD available.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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