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

Recruiting now · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Neurotrophic Keratitis

In brief

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.

Key facts

Study ID

NCT06999733

Run by

Krystal Biotech, Inc.

People needed

60

Starts

2025-07-07

Expected to finish

2027-12-01

Last updated by the study team

2026-02-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Aged ≥ 18 years at the time of informed consent.
• Diagnosis of Stage 2 or Stage 3 NK, as defined by the Mackie criteria.
• 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).
• PCED at Screening 1 must have a minimum of 1 mm of any linear measurement of defect.
• A negative pregnancy test at Screening 1 and Day 1 for women of child-bearing potential.

You may not qualify if…

• 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.
• 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.
• Anticipated use of therapeutic contact lenses or contact lens wear for refractive correction during the study Treatment Period in the study eye.
• 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.
• PCED at Screening 1 or 2 of > 8 mm maximum linear measurement in any eye.
• The central 3 mm of corneas contain PCED bilaterally at Screening 1 or 2.
• Anticipated need for punctal occlusion of the study eye during the Treatment Period.
• 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).
• 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).
• Intraocular surgery on the eligible eye(s) within 6 months prior to Screening 1, and any ophthalmic condition that may require surgery on the study eye during the Treatment Period. An exception to the preceding statement is allowed if, in the opinion of the Investigator, the ocular surgery is deemed the cause of the NK.
• Corneal transplantation (i.e., keratoplasty) of the study eye within the past 2 years or a history of multiple keratoplasty operations to the study eye regardless of timing. This includes full- or partial-thickness, or endothelial keratoplasty.
• Subject who is pregnant or nursing.
• Subject who is unwilling to comply with contraception requirements per-protocol (for subjects of childbearing potential).
• Subject who is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.

Where it is running

• Vance Thompson Vision — West Fargo, North Dakota, United States (enrolling)
• Azul Vision - California Eye Specialists Medical Group Inc. — Pasadena, California, United States (enrolling)
• Midwest Cornea Associates — Carmel, Indiana, United States (enrolling)
• Vance Thompson Vision — Alexandria, Minnesota, United States (enrolling)
• Minnesota Eye Consultants — Minnetonka, Minnesota, United States (enrolling)
• St. Louis Eye Institute — Town and Country, Missouri, United States (enrolling)
• Metropolitan Eye Research and Surgery Institute — Palisades Park, New Jersey, United States (enrolling)
• UPMC Vision Institute — Pittsburgh, Pennsylvania, United States
• UC Irvine Alpha Clinic — Irvine, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.