Journal article

Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery

Ravi Savarirayan, Melita Irving, Wirginia Maixner, Dominic Thompson, Amaka C Offiah, Daniel JA Connolly, Ashok Raghavan, James Powell, Marcin Kronhardt, George Jeha, Sajda Ghani, Elena Fisheleva, Jonathan RS Day



Achondroplasia causes narrowing of the foramen magnum and the spinal canal leading to increased mortality due to cervicomedullary compression in infants and significant morbidity due to spinal stenosis later in adulthood. Vosoritide is a C-natriuretic peptide analogue that has been shown to improve endochondral ossification in children with achondroplasia. The objective of this trial is to evaluate the safety of vosoritide and whether vosoritide can improve the growth of the foramen magnum and spinal canal in children that may require decompression surgery. An Achondroplasia Foramen Magnum Score will be used to identify infants at risk of requiring decompression surgery. This is a 2-year ope..

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Funding Acknowledgements

The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: Funded by BioMarin Pharmaceutical.