VIrtual STudy in Achondroplasia for the US (VISTA) Study Protocol
VISTA
What is the goal of the study?
Achondroplasia (ACH) is a rare, inherited, autosomal dominant condition with a worldwide prevalence of approximately 4 in100,000. A key clinical feature of ACH is severe short stature with disproportionate growth manifested in babies as a long narrow trunk and shortening of the proximal part of the arms and legs.In addition to disproportionate short stature that leads to functional limitations and has detrimental impact on activities of daily living, the abnormal bone growth in ACH is associated with multiple medical complications that cause considerable morbidity. Voxzogo® (vosoritide) is a commercially available treatment for children with ACH. BioMarin, the study sponsor, has partnered with PicnicHealth to generate real-world data to contextualize the impact of Voxzogo® While the efficacy of Voxzogo® has been established in a clinical trial setting, there remains a need to demonstrate the response of individuals with ACH to Voxzogo® in the real-world. Furthermore, there are numerous scientific questions remaining to be answered in terms of how a gain in height may translate into other tangible individual benefits over the long term. This study will provide insight into the real world experience of patients treated with Voxzogo®. It is also important to generate additional data on untreated individuals with ACH to elucidate the value and effectiveness of Voxzogo® treatment in the real-world. Additionally, the results of this study may be published and will provide the community with a collection of data that highlights key findings such as any observed differences in those who receive therapeutic intervention vs those who do not.
Who can participate in the study?
Please contact the study team listed below to learn more.