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

Kyowa Kirin

Status and phase: Enrolling; Phase 3

Conditions: Achondroplasia

Treatments: Drug: KK8398

Study type: Interventional

Funder types: Industry

Identifiers

NCT06926491

jRCT2031240562 (Registry Identifier)

8398-001

Details and patient eligibility

About

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

Enrollment: 6 estimated patients

Sex: All

Ages: 3 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

KK8398

Experimental group

Treatment:

Drug: KK8398