New pill aims to slow vision loss in rare usher syndrome

NCT ID NCT04355689

First seen Jan 05, 2026 · Last updated May 15, 2026 · Updated 16 times

Summary

This study tests whether an experimental tablet called NPI-001 can safely slow or stop vision loss in people with Usher syndrome, a rare genetic condition that causes blindness and hearing loss. About 49 adults will take either the drug or a placebo for 24 months. The main goal is to see if the drug helps preserve the retina's ability to sense light.

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Contacts and locations

Locations

  • CERA

    Melbourne, Australia

  • Lions Eye Institute

    Perth, Australia

  • Queensland Eye Institute

    Brisbane, Australia

  • Sydney Eye Hospital / Save Sight Institute

    Sydney, Australia

Conditions

Explore the condition pages connected to this study.