What is KB803 studying for dystrophic epidermolysis bullosa?

KB803 is being studied to see if it is safe and effective in reducing corneal abrasion symptoms in patients with dystrophic epidermolysis bullosa. This trial compares KB803 eye drops to a placebo and is sponsored by Krystal Biotech, Inc. Participants will receive treatments at home and be monitored for safety and symptom changes.

Who may be eligible for this dystrophic epidermolysis bullosa clinical trial?

Participants must be at least 6 months old with a confirmed diagnosis of DEB involving the COL7A1 gene mutation. Both males and females can participate, but pregnant or nursing women are excluded. Participants should have frequent corneal abrasions and be able to follow study procedures. Please review eligibility criteria for more details.

Is there any cost to participate in this dystrophic epidermolysis bullosa clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any costs incurred during participation.

Actively Recruiting

Phase 3

Age: 6Months +

All Genders

ID07016750

A Phase 3 Double-Blind Study Comparing KB803 Eye Treatment to Placebo for Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa

Led by Krystal Biotech, Inc. · Updated on 2026-03-12

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating KB803, an ophthalmic suspension containing a modified herpes simplex virus expressing human collagen VII protein, in patients with dystrophic epidermolysis bullosa DEB who experience recurrent corneal abrasions. This Phase 3, double-blind, randomized, placebo-controlled crossover study aims to assess the safety and effectiveness of KB803 compared to a matched placebo in both children and adults with DEB. The study is sponsored by Krystal Biotech, Inc. Participants who have been in the Sponsors natural history study for at least 12 weeks and meet other criteria will be randomly assigned to receive either KB803 or placebo eye drops three times a week for the first 12 weeks. Then, they will switch to the alternate treatment for another 12 weeks. The eye treatments are given at home by a trained person, with monitoring by the study team. During the 24-week study, researchers will monitor safety and tolerability, track the frequency of corneal abrasion symptoms, and assess eye pain. Participants or their guardians will keep weekly symptom diaries and complete monthly questionnaires about symptoms and disease severity. Study staff will regularly check for any side effects or changes in treatment. The total participation lasts 24 weeks with ongoing safety and symptom evaluations.

CONDITIONS

Brief Title

A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa

Who Can Participate

Age: 6Months +

All Genders

Eligibility Criteria

You may qualify if you...

The subject and/or their parent/legal guardian must provide informed consent/assent and be willing to follow study procedures and instructions.
Age 6 months or older at time of informed consent/assent.
Confirmed diagnosis of dystrophic epidermolysis bullosa (DEB) with a mutation in the COL7A1 gene.
Meets minimum corneal abrasion symptom frequency in the natural history study.

You will not qualify if you...

Starting any new treatment or changing treatment for ocular disease during the run-in period except preservative-free topical antibiotics or artificial tears/lubricants.
Treatment with an investigational agent or off-label ophthalmic use during the run-in period or planned use during the study without medical monitor approval.
Any condition that would affect study assessments, interfere with drug administration, or pose additional risk.
Women who are pregnant or nursing.
Unwillingness to comply with contraception requirements per protocol.
Known or likely noncompliance with study protocol requirements in the opinion of the Investigator.

Research Team

David Sweet, MD, PhD

Brittani Agostini, RN, CCRC

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