Actively Recruiting
A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
Led by Krystal Biotech, Inc. · Updated on 2026-03-12
16
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
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 Sponsor's 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
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.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive ophthalmic administration of KB803 or placebo three times a week at home, with a crossover to the alternative treatment after 12 weeks.
Weekly contact for adverse event assessment; weekly symptom diaries and monthly questionnaires completed by participants or guardians
Trial Site Locations
Total: 5 locations
1
Mission Dermatology Center
Rancho Santa Margarita, California, United States, 92688
Actively Recruiting
2
Skin Research Institute
Coral Gables, Florida, United States, 33146
Actively Recruiting
3
UMass Memorial Medical Center
Worcester, Massachusetts, United States, 01605
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
5
Texas Dermatology and Laser Specialists
San Antonio, Texas, United States, 78218
Actively Recruiting
Research Team
D
David Sweet, MD, PhD
B
Brittani Agostini, RN, CCRC
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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