Achondroplasia
Conditions
Interventions
Navepegritide will be administered subcutaneously once weekly at a dose of 100 microg/kg.
The duration of treatment will be 52 weeks.
Sponsors
Fujita Mariko
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Male or female, between 2 and 11 years of age (inclusive) at the time of informed consent. -Have been genetically confirmed as ACH and have a clinical diagnosis at the time of screening test (documentation of past test results is available as proof of diagnosis). -Able to stand without assistance. -Parent(s) or legal guardian(s) are willing to comply with the protocol and able to administer once-weekly subcutaneous injections of investigational product. -Treatment with vosoritide at the time of screening test which is continued for the last >=26 weeks (for patients previously treated with somatropin, at least 12 months must have passed between the last dose of somatropin and the time of screening test).
Exclusion criteria
Exclusion criteria: -Participation in an interventional clinical study within 3 months prior to screening test. -Radiographically confirmed epiphyseal closure or a low annualized growth velocity (AGV =450 msec on 12-lead electrocardiogram. -Known history or presence of malignant disease.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in ACH-specific height Z-score (Z-score using the growth curve of untreated ACH patients as the reference) from baseline to Week 52 | — |
Contacts
Public ContactMariko Fujita
Teijin Pharma Limited
Outcome results
None listed