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RECRUITINGPHASE1/PHASE2

A Study Comparing KB801 Verse Placebo in Patients With Stage 2 or 3 Neurotrophic Keratitis

A Phase 1/2, Multicenter, Double-Masked, Placebo-Controlled Study of KB801 in Subjects With Stage 2 or 3 Neurotrophic Keratitis

NCT06999733•Krystal Biotech, Inc.

View on ClinicalTrials.gov

Summary

The Sponsor is developing KB801, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human nerve growth factor (NGF) to the corneas of people with Stage 2 or 3 neurotrophic keratitis (NK) via topical application. This is a Phase 1/2, multicenter, double-masked, placebo-controlled study to evaluate the safety, tolerability, and preliminary efficacy of KB801 in these subjects.

Detailed Description

Subjects will will be randomized 1:1 to receive KB801 or placebo (vehicle) topically to the study eye daily for 8 weeks. Subjects will return for a follow-up visit 2- weeks after finishing treatment to monitor for safety and durability of corneal healing. Safety follow-up visits will then occur every 3 months through approximately 1 year on study.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. The subject must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions.
•2. Aged ≥ 18 years at the time of informed consent.
•3. Diagnosis of Stage 2 or Stage 3 NK, as defined by the Mackie criteria.
•4. Persistent corneal epithelial defect (PCED) between Screening 1 and Screening 2 without clinical improvement during that time. PCED is defined as a focal loss of the cornea's outermost cell layer and can include involvement of the deeper stromal layers (i.e., corneal ulcer).
•5. PCED at Screening 1 must have a minimum of 1 mm of any linear measurement of defect.
•6. A negative pregnancy test at Screening 1 and Day 1 for women of child-bearing potential.

Exclusion Criteria

•1. An active ocular infection (bacterial, viral, fungal or protozoal) or active ocular inflammation not related to NK in the study eye as assessed by Investigator at Screening 1 or 2.
•2. Use of any prohibited therapies at Screening or anticipated need for topical treatments other than the investigational product or products allowed per protocol in the study eye from Screening through the study Treatment Period
•a. Sufficient washout of prohibited therapies must occur prior to meeting eligibility criteria.
•3. Anticipated use of therapeutic contact lenses or contact lens wear for refractive correction during the study Treatment Period in the study eye.
•4. Evidence of corneal ulceration involving the posterior third of the corneal stroma, corneal melting, descemetocele, impending or perforation in the study eye at the time of Screening 1 or 2.
•5. PCED at Screening 1 or 2 of \> 8 mm maximum linear measurement in any eye.
•6. The central 3 mm of corneas contain PCED bilaterally at Screening 1 or 2.
•7. Anticipated need for punctal occlusion of the study eye during the Treatment Period.
•8. Presence or history of any ocular or systemic disorder or condition that might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the Investigator to be incompatible with the study visit schedule or conduct (e.g., progressive or degenerative corneal or retinal conditions, uveitis, optic neuritis, poorly controlled diabetes, autoimmune disease, systemic infection, neoplastic diseases).
•9. Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to Day 1 (participation in a non-interventional observational study is permitted).
+5 more criteria

Locations (9)

UC Irvine Alpha Clinic

Irvine, California, United States

Azul Vision - California Eye Specialists Medical Group Inc.

Pasadena, California, United States

Midwest Cornea Associates

Carmel, Indiana, United States

Vance Thompson Vision

Alexandria, Minnesota, United States

Minnesota Eye Consultants

Minnetonka, Minnesota, United States

St. Louis Eye Institute

Town and Country, Missouri, United States

Metropolitan Eye Research and Surgery Institute

Palisades Park, New Jersey, United States

Vance Thompson Vision

West Fargo, North Dakota, United States

UPMC Vision Institute

Pittsburgh, Pennsylvania, United States

Key Dates

Start Date

July 7, 2025

Primary Completion Date

December 1, 2026

Completion Date

December 1, 2027

Last Updated

February 27, 2026

Enrollment

ESTIMATED participants

Conditions

Neurotrophic Keratitis

Interventions

KB801

BIOLOGICAL

Placebo

DRUG

Contacts

David Sweet, MD, PhD

CONTACT

412-586-5830 dsweet@krystalbio.com

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830 bagostini@krystalbio.com

OCT Evaluation of Neurotrophic Ulcer Following Treatment With Oxervate

NCT04573647

Neurotrophic UlcerNeurotrophic Keratitis+2 more

View study

COMPLETEDPHASE2

REC 0/0559 Eye Drops for Treatment of Moderate and Severe Neurotrophic Keratitis in Adult Patients

NCT04276558

Neurotrophic Keratitis

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Study Comparing KB801 Verse Placebo in Patients With Stage 2 or 3 Neurotrophic Keratitis

Inclusion Criteria

Exclusion Criteria

OCT Evaluation of Neurotrophic Ulcer Following Treatment With Oxervate

REC 0/0559 Eye Drops for Treatment of Moderate and Severe Neurotrophic Keratitis in Adult Patients

Corneal Nerves After Treatment With Cenegermin

Open-label Trial to Evaluate Efficacy and Safety of rhNGF on Corneal Thickness Via AS-OCT in Neurotrophic Keratitis

Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Neurotrophic Keratitis Subjects

Evaluation of Safety and Efficacy of rhNGF in Patients With Stage 2 and 3 Neurotrophic Keratitis.