NCT06999733

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Neurotrophic Keratitis

Interventions

Type	Name	Description
BIOLOGICAL	KB801	Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector delivering functional, full-length human nerve growth factor (NGF)
DRUG	Placebo	Vehicle

Timeline

• Start date: 2025-07-07
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2025-05-31
• Last updated: 2026-02-27

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study