Experimental drug aims to fix faulty kidney protein in rare genetic disease

NCT ID NCT05448755

Summary

This small, early-stage study tested whether daily injections of an experimental drug called ELX-02 are safe and can improve kidney health in adults with a specific genetic form of Alport Syndrome. The drug is designed to help the body produce a crucial protein that is missing due to a genetic error. Researchers measured changes in kidney structure and protein in the urine over 8 weeks of treatment.

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Contacts and locations

Locations

  • Great Ormond Street Hospital

    London, WC1N 3JH, United Kingdom

  • Monash Medical Center

    Clayton, Victoria, 3168, Australia

  • Royal Children's Hospital

    Parkville, Victoria, 3051, Australia

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

Conditions

Explore the condition pages connected to this study.