Achondroplasia Clinical Trial
Official title:
A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States
This is an observational study of a pediatric cohort with achondroplasia in the United States. This cohort consists of both individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats: Participant-mediated access to electronic health records which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data The primary study population will include individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa)
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