Gene therapy eye drops aim to heal corneas in rare nerve disorder

NCT ID NCT06999733

Recruiting now Disease control Sponsor: Krystal Biotech, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a new gene therapy called KB801 for people with stage 2 or 3 neurotrophic keratitis, a rare eye disease where the cornea cannot heal itself. The treatment uses a harmless virus to deliver nerve growth factor directly to the eye. About 60 participants will receive either KB801 or a placebo to see if it safely helps close persistent corneal defects.

What this could mean

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

Active substance: KB801 (a modified herpes virus that delivers nerve growth factor to the eye)
What this could lead to: If it works, this could provide a new treatment to heal persistent corneal damage in people with neurotrophic keratitis.
What could go wrong: This is an early phase 1/2 trial with only 60 participants, so results may not apply to everyone. The treatment uses a modified virus, which could cause side effects or not work as expected.

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Conditions

The condition(s) this trial relates to.

neurotrophic keratopathy

As listed by the trial registrant

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

NEUROTROPHIC KERATITIS

Contacts and locations

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Locations

Azul Vision - California Eye Specialists Medical Group Inc.
RECRUITING

Pasadena, California, 91107, United States
Metropolitan Eye Research and Surgery Institute
RECRUITING

Palisades Park, New Jersey, 07650, United States
Midwest Cornea Associates
RECRUITING

Carmel, Indiana, 46290, United States

Contact Email: •••••@•••••
Minnesota Eye Consultants
RECRUITING

Minnetonka, Minnesota, 55305, United States
St. Louis Eye Institute
RECRUITING

Town and Country, Missouri, 63017, United States
UC Irvine Alpha Clinic
NOT_YET_RECRUITING

Irvine, California, 92617, United States
UPMC Vision Institute
NOT_YET_RECRUITING

Pittsburgh, Pennsylvania, 15219, United States
Vance Thompson Vision
RECRUITING

Alexandria, Minnesota, 56308, United States
Vance Thompson Vision
RECRUITING

West Fargo, North Dakota, 58078, United States

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