臨床研究等提出・公開システム

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

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

Sato Yu

Kyowa Kirin Co., Ltd.

1-9-2, Otemachi , Chiyoda-ku, Tokyo

yu.sato.1t@kyowakirin.com

Clinical trial information contact

Kyowa Kirin Co., Ltd.

1-9-2, Otemachi , Chiyoda-ku, Tokyo

+81-3-5205-7200

clinical.info.jp@kyowakirin.com

Recruiting

May. 01, 2025

Interventional

non-randomized controlled trial

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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.

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.

Both

Achondroplasia

After obtaining written consent, patients with achondroplasia will have their previous treatment(s) washed out if necessary, and then begin treatment with KK8398.

Change from Baseline in annualized height velocity

Change from Baseline at each evaluation point in annualized height velocity

Dec. 12, 2024

Yes

The datasets generated and/or analyzed during the study sponsored by Kyowa Kirin will be available in the Vivli repository, https://vivli.org/ourmember/kyowa-kirin/ as long as conditions of data disclosure specified in the policy section of the Vivli website are satisfied.

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