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Can an antioxidant pill slow blindness in usher syndrome?

NCT ID NCT07710196

Not yet recruiting Disease control Sponsor: Nacuity Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jul 17, 2026 · Last updated Jul 17, 2026

Summary

This phase 3 trial tests whether an experimental drug called NPI-001 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 take either NPI-001 or a placebo twice daily for 24 months. Researchers measure changes in the area of the retina's ellipsoid zone to see if the drug preserves vision.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
an experimental oral drug called NPI-001 (N-acetyl cysteine amide)
What this could lead to
If it works, this could point toward a treatment that slows vision loss in people with Usher syndrome.
What could go wrong
This is a relatively small trial, and many promising drugs fail to show benefit in larger studies. The drug may not slow vision loss or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

RETINITIS PIGMENTOSA (RP) USHER SYNDROME

Contacts and locations

Study contacts

  • Contact

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

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