A Study Comparing KB801 Verse Placebo in Patients With Stage 2 or 3 Neurotrophic Keratitis
EMERALD-1
A Phase 1/2, Multicenter, Double-Masked, Placebo-Controlled Study of KB801 in Subjects With Stage 2 or 3 Neurotrophic Keratitis
1 other identifier
interventional
60
1 country
9
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Jul 2025
Typical duration for phase_1
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 14, 2025
CompletedFirst Posted
Study publicly available on registry
May 31, 2025
CompletedStudy Start
First participant enrolled
July 7, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2027
February 27, 2026
February 1, 2026
1.4 years
May 14, 2025
February 25, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
To evaluate safety and tolerability of KB801 based upon assessment of adverse events (frequency, severity, relatedness).
Number of subjects with treatment related adverse events as assessed by CTCAE v5.
8 weeks
To evaluate the effect of KB801 on complete healing of the Persistent Corneal Epithelial Defect (PCED) in subjects with Stage 2 or Stage 3 NK
Proportion of subjects receiving KB801 exhibiting complete PCED closure at Week 8 compared to those receiving placebo.
8 weeks
Secondary Outcomes (2)
To evaluate the effect of KB801 on complete healing of the PCED in subjects with Stage 2 or 3 NK
10 weeks
To evaluate the effect of KB801 on conventional healing of the PCED in subjects with Stage 2 or 3 NK
10 weeks
Study Arms (2)
KB801
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
UC Irvine Alpha Clinic
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
St. Louis Eye Institute
Town and Country, Missouri, 63017, United States
Metropolitan Eye Research and Surgery Institute
Palisades Park, New Jersey, 07650, United States
Vance Thompson Vision
West Fargo, North Dakota, 58078, United States
UPMC Vision Institute
Pittsburgh, Pennsylvania, 15219, United States
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 14, 2025
First Posted
May 31, 2025
Study Start
July 7, 2025
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
December 1, 2027
Last Updated
February 27, 2026
Record last verified: 2026-02