First ALS patients receive experimental gene therapy in groundbreaking safety trial

NCT ID NCT07401121

Summary

This early-stage study is testing the safety of a new gene therapy called CTx1000 in people with ALS. The therapy aims to target a protein called TDP-43 that builds up in ALS. Researchers will give a single dose to 15 participants and closely monitor for side effects while also checking if it might help slow disease progression.

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Contacts and locations

Locations

  • Macquarie University Hospital

    RECRUITING

    Sydney, New South Wales, 2109, Australia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.