Achondroplasia
Conditions
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
Interventions
DRUGKK8398
KK8398 will be administered.
Sponsors
Kyowa Kirin Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in annualized height velocity | Pre-dose, Week 52 |
Secondary
| Measure | Time frame |
|---|---|
| Height Z-score using growth tables in patients with achondroplasia and the change from baseline | Pre-dose, Week 52 |
Countries
Japan
Contacts
Primary ContactKyowa Kirin Co., Ltd.
Outcome results
None listed