New drug aims to preserve sight in rare genetic blindness

NCT ID NCT07290530

First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 1 time

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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa Usher syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.