Daily injection trial aims to help babies with dwarfism avoid risky spinal surgery

NCT ID NCT04554940

Summary

This study tests the safety of daily vosoritide injections in infants with achondroplasia (the most common form of dwarfism) who are at risk of needing spinal decompression surgery. Researchers want to see if the treatment is safe for very young children and whether it affects skull growth around the spinal cord. The trial includes 20 infants under 12 months old who show signs that might require surgery.

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Contacts and locations

Locations

  • Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

    London, SE1 7EH, United Kingdom

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH, United Kingdom

Conditions

Explore the condition pages connected to this study.