RECRUITING

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

Conditions

Neurotrophic Keratitis Persistent Corneal Epithelial Defect Corneal Epithelial Defect

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2025-05

Study Completion:2026-10

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06999733

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Contact

David Sweet, MD, PhD

CONTACT

412-586-5830

dsweet@krystalbio.com

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830

bagostini@krystalbio.com

Study Locations (Sites)

University of California Irvine

Irvine, California, 92617

United States

Azul Vision - California Eye Specialists Medical Group Inc.

Pasadena, California, 91107

United States

Midwest Cornea Associates

Carmel, Indiana, 46290

United States

Vance Thompson Vision

Alexandria, Minnesota, 56308

United States

Minnesota Eye Consultants

Minnetonka, Minnesota, 55305

United States

UPMC Vision Institute

Pittsburgh, Pennsylvania, 15219

United States

Collaborators and Investigators

Sponsor: Krystal Biotech, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-05

Study Completion Date2026-10

Study Record Updates

Study Start Date2025-05

Study Completion Date2026-10

Terms related to this study

Keywords Provided by Researchers

Neurotrophic Keratitis
Persistent Corneal Epithelial Defect
Corneal Epithelial Defect

Additional Relevant MeSH Terms

Neurotrophic Keratitis