NCT07016750

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

A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

Summary

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Detailed description

Subjects participating in the Sponsor's natural history study (protocol NHS) for at least 12 weeks and that meet all other eligibility criteria will be randomized 1:1 to receive ophthalmic administration of B-VEC three times a week or placebo three times a week for the first 12 week intervention period. During the second 12-week intervention period subjects will be crossed-over to receive the alternative Investigational Product (IP) for an additional 12 weeks. IP will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Subjects (or their parent/legal guardian) will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.

Conditions

• Dystrophic Epidermolysis Bullosa
• DEB - Dystrophic Epidermolysis Bullosa
• Recessive Dystrophic Epidermolysis Bullosa
• Dominant Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	KB803	Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein
DRUG	Placebo	Vehicle

Timeline

• Start date: 2025-06-20
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2025-06-12
• Last updated: 2026-03-12

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study