New drug aims to preserve sight in rare genetic blindness

NCT ID NCT07290530

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This phase 3 trial tests whether NPI-001, taken twice daily for 24 months, can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants will receive either the drug or a placebo. The study measures changes in the area of healthy retina over time and tracks any side effects.

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