KB801 for Keratitis

(EMERALD-1 Trial)

Recruiting at 5 trial locations

Overseen By Brittani Agostini, RN, CCRC

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Krystal Biotech, Inc.

Must not be taking: Topical treatments

Disqualifiers: Active ocular infection, Corneal ulceration, Diabetes, Autoimmune disease, others

Compensation: Varies

Number of Visits: 2

Duration: 8 weeks

27 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called KB801 for individuals with neurotrophic keratitis (NK), a condition affecting the cornea of the eye. The goal is to determine if KB801 is safe and effective in promoting corneal healing by delivering a nerve growth factor directly to the eye. Participants will receive either the experimental treatment or a placebo (a substance with no active effect) for comparison. This trial may suit those with Stage 2 or 3 NK who have had a persistent corneal defect for at least two weeks without improvement. As a Phase 1, Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial requires that you stop using certain medications before participating, as there is a need for a 'washout period' (time without taking certain medications) for prohibited therapies. You should discuss your current medications with the study team to see if they are allowed.

Is there any evidence suggesting that KB801 is likely to be safe for humans?

Research has shown that KB801 is still undergoing testing to determine its safety and effectiveness for treating neurotrophic keratitis (NK), an eye condition. As this is an early-stage study, the primary goal is to assess the safety and tolerability of KB801. Currently, limited information is available about side effects or adverse reactions.

Researchers closely monitor for any safety issues due to the trial's early stage. Early trials primarily focus on safety to ensure the treatment does not cause harm. As more data emerges, it will provide a clearer understanding of KB801's safety profile. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for keratitis, which often involve antibiotics or antiviral medications, KB801 is unique because it targets the underlying causes of inflammation directly. Most current treatments focus on managing symptoms or eradicating infectious agents, but KB801 works by modulating immune responses to reduce inflammation more effectively. Researchers are excited about KB801 because it has the potential to provide relief faster and with fewer side effects than traditional therapies, offering a novel approach to treating keratitis.

What evidence suggests that KB801 might be an effective treatment for neurotrophic keratitis?

Research shows that KB801, which participants in this trial may receive, is a new treatment for neurotrophic keratitis (NK), a condition where the cornea loses feeling and can't heal properly. This treatment uses a safe virus that neither multiplies nor alters DNA. It delivers nerve growth factor (NGF) directly to the cornea through eye drops. NGF plays a crucial role in repairing nerve damage, which is essential in NK. Early results suggest that this method could help heal the cornea by restoring nerve function and improving eye health. While more information is needed, the scientific basis for KB801 appears promising. ^{2 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Stage 2 or 3 Neurotrophic Keratitis, a condition affecting the cornea. Participants must have persistent corneal defects and decreased sensitivity but no active infections or inflammation unrelated to NK. They should not be pregnant, nursing, or unwilling to use contraception and must agree to follow study procedures.

Inclusion Criteria

A negative pregnancy test at Screening 1 and Day 1 for women of child-bearing potential

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

PCED at Screening 1 must have a minimum of 1 mm of any linear measurement of defect

Exclusion Criteria

I do not have an active eye infection or inflammation unrelated to my condition.

I haven't used any disallowed treatments for my condition.

PCED at Screening 1 or 2 of > 8 mm maximum linear measurement in any eye

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive KB801 or placebo topically to the study eye twice weekly for 8 weeks

8 weeks

Follow-up

Participants are monitored for safety and durability of corneal healing after treatment

12 weeks

Follow-up visits at 2 and 12 weeks after treatment, then every 3 months up to 1 year

What Are the Treatments Tested in This Trial?

Interventions

• KB801

Trial Overview KB801, a new treatment using a modified herpes virus to deliver nerve growth factor to the eye's surface, is being tested against a placebo in people with advanced neurotrophic keratitis. This double-masked study means neither participants nor researchers know who receives the real treatment.

How Is the Trial Designed?

Group I: KB801 Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Krystal Biotech, Inc.

Lead Sponsor

Trials

15

Recruited

770+

Citations

1. ir.krystalbio.com ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-first-patient-dosed-phase-12-trial

Krystal Biotech Announces First Patient Dosed in Phase 1/ ... KB801 is a redosable eye drop gene therapy in development for the treatment of NK, a degenerative corneal disease characterized by damage or loss of function ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06999733

A Study Assessing KB801 for the Treatment of Stage 2 or 3 ... A Study Assessing KB801 for the Treatment of Stage 2 or 3 Neurotrophic Keratitis (EMERALD-1). ClinicalTrials.gov ID NCT06999733.

3. ophthalmologytimes.com ophthalmologytimes.com/view/krystal-biotech-doses-first-patient-in-emerald-1-phase-1-2-trial-for-neurotrophic-keratitis

Krystal Biotech doses first patient in EMERALD-1 phase ... Key Takeaways. EMERALD-1 trial evaluates KB801, a gene therapy for neurotrophic keratitis, focusing on safety and efficacy in corneal healing.

4. ir.krystalbio.com ir.krystalbio.com/static-files/bb0eb26b-2d44-4dd3-bdad-8531bff06d87

KB801 for Neurotrophic Keratitis First Patient Dosed Update KB801 being relatively short and cost-effective with safety, efficacy, and CMC ... KB803 and KB801, respectively; the timing of data readouts from the ...

5. globenewswire.com globenewswire.com/news-release/2025/07/09/3112425/0/en/Krystal-Biotech-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-of-KB801-for-the-Treatment-of-Neurotrophic-Keratitis.html

Krystal Biotech Announces First Patient Dosed in Phase 1/2 Based on available claims data, an estimated 68,000 patients in the United States had a NK claim in 2024, up over 115% from 31,000 patients with ...

6. ir.krystalbio.com ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-platform-technology-designation-hsv

Krystal Biotech Receives FDA Platform Technology ... The safety and efficacy of KB801 for the treatment of NK are currently under evaluation in EMERALD-1, the Company's 2:1 randomized, double ...

7. modernod.com modernod.com/news/krystal-biotech-announces-first-patient-dosed-in-phase-12-trial-of-kb801-for-the-treatment-of-neurotrophic-keratitis/2482883/

Krystal Biotech Announces First Patient Dosed in Phase 1/ ... The primary objective of EMERALD-1 is to evaluate the safety and tolerability of topical ocular administration of KB801 in patients with NK. The ...