Evaluating KK8398 for treating achondroplasia in children and adolescents

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

This study is testing if a new medication called KK8398 can help children and teens with achondroplasia grow taller over a year.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 3 Years to 18 Years
• Sex: All
• Sponsor: Kyowa Kirin Co., Ltd. (Industry-sponsored)
• Locations: 9 sites (Ōbu, Aichi-ken and 8 other locations)
• Trial ID: NCT06926491 on ClinicalTrials.gov

What this trial studies

This trial aims to assess the efficacy and safety of KK8398 in improving annualized height velocity in patients diagnosed with achondroplasia. Participants aged between 2.5 and 17.5 years will receive repeated doses of KK8398 over a 52-week period. The study will monitor growth outcomes and any potential side effects associated with the treatment. The trial is designed to provide insights into the effectiveness of KK8398 as a therapeutic option for this condition.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 2.5 to 17.5 years diagnosed with achondroplasia through genetic testing.

Not a fit: Patients who are already at a certain height threshold for their age and gender or have received specific prior treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly enhance growth and quality of life for children with achondroplasia.

How similar studies have performed: While this approach is novel in the context of KK8398, similar studies targeting growth in achondroplasia have shown promise.

Eligibility criteria

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.

Where this trial is running

Ōbu, Aichi-ken and 8 other locations

• 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)

Study contacts

• Study coordinator: Kyowa Kirin Co., Ltd.

Conditions Achondroplasia