A Study Assessing KB801 for the Treatment of Stage 2 or 3 Neurotrophic Keratitis

NCT ID: NCT06999733

Last Updated: 2025-05-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-31

Study Completion Date

2026-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Subjects will will be randomized 2:1 to receive KB801 or placebo (vehicle) topically to the study eye twice weekly for 8 weeks. Subjects will return for follow-up visits 2- and 12-weeks after finishing treatment to monitor for safety and durability of corneal healing. Safety follow-up visits will then occur every 3 months up to 1 year on study.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Neurotrophic Keratitis

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Neurotrophic Keratitis Persistent Corneal Epithelial Defect Corneal Epithelial Defect

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

KB801

Group Type EXPERIMENTAL

KB801

Intervention Type BIOLOGICAL

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)

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Vehicle

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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)

Intervention Type BIOLOGICAL

Placebo

Vehicle

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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) of at least 2 weeks duration 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. Decreased corneal sensitivity (≤ 4 cm using the Cochet-Bonnet esthesiometer \[CBE\]) within the area of the PCED and in at least one corneal quadrant outside of the area of the defect.
7. 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

1. The study Treatment Period is defined as Study Days 1 through 53
2. 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).
10. 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.
11. Corneal transplantation (i.e., keratoplasty) of the study eye. This includes full- or partial-thickness, or endothelial keratoplasty.
12. Subject who is pregnant or nursing.
13. Subject who is unwilling to comply with contraception requirements per-protocol (for subjects of childbearing potential).
14. 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Krystal Biotech, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of California Irvine

Irvine, California, United States

Site Status NOT_YET_RECRUITING

Azul Vision - California Eye Specialists Medical Group Inc.

Pasadena, California, United States

Site Status NOT_YET_RECRUITING

Midwest Cornea Associates

Carmel, Indiana, United States

Site Status NOT_YET_RECRUITING

Vance Thompson Vision

Alexandria, Minnesota, United States

Site Status RECRUITING

Minnesota Eye Consultants

Minnetonka, Minnesota, United States

Site Status RECRUITING

UPMC Vision Institute

Pittsburgh, Pennsylvania, United States

Site Status NOT_YET_RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

David Sweet, MD, PhD

Role: CONTACT

Phone: 412-586-5830

Email: [email protected]

Brittani Agostini, RN, CCRC

Role: CONTACT

Phone: 412-586-5830

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

UC Irvine Alpha Clinic

Role: primary

Kaci Taylor

Role: primary

Chris Bartizal, BS

Role: primary

Miranda Danielson

Role: primary

Bailee Larson

Role: backup

Allison Dreyer

Role: primary

UPMC Vision Institute Clinical Trials Team

Role: primary

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

KB801-01

Identifier Type: -

Identifier Source: org_study_id