KB803 eye drops versus placebo for people 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
This trial tests whether KB803 eye drops, given three times a week, can reduce recurrent corneal abrasions in children and adults with dystrophic epidermolysis bullosa.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 16 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Krystal Biotech, Inc. Industry-sponsored |
| Locations | 5 sites (Rancho Santa Margarita, California and 4 other locations) |
| Trial ID | NCT07016750 on ClinicalTrials.gov |
What this trial studies
This is a Phase 3, double-blind, randomized, placebo-controlled crossover trial enrolling pediatric and adult participants with DEB and recurrent corneal abrasions. Eligible participants already in the sponsor's natural history study for at least 12 weeks will be randomized 1:1 to receive ophthalmic KB803 or matched placebo three times weekly for 12 weeks, then crossed over to the alternate treatment for an additional 12 weeks. Dosing is administered at home by a trained designee, with investigators monitoring safety via contacts for adverse events and changes in treatments. Participants complete weekly symptom diaries and monthly questionnaires to record corneal abrasion frequency, symptoms, and overall impressions of disease change.
Who should consider this trial
Good fit: Children (6 months and older) and adults with genetically confirmed DEB caused by COL7A1 mutations who have recurrent corneal abrasions and have participated in the sponsor's natural history study for at least 12 weeks.
Not a fit: People without recurrent corneal abrasions, those with other ocular conditions or recent changes in ocular treatments, or those using other investigational ophthalmic agents are unlikely to benefit from this specific intervention.
Why it matters
Potential benefit: If successful, KB803 could reduce the frequency and severity of corneal abrasions and improve eye comfort and visual function for people with DEB.
How similar studies have performed: Vector-based and topical therapies for DEB skin lesions have shown encouraging results in earlier studies, but ophthalmic application for corneal abrasions is a relatively new approach with limited prior data.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions. 2. Age 6 months or older at time of informed consent/assent. 3. Confirmed diagnosis of DEB with a mutation in the COL7A1 gene. 4. Meets minimum corneal abrasion symptom frequency in the NHS study. Exclusion Criteria: 1. Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions. 2. Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis). 3. Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject. 4. Women who are pregnant or nursing. 5. Subject who is unwilling to comply with contraception requirements per protocol. 6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.
Where this trial is running
Rancho Santa Margarita, California and 4 other locations
- Mission Dermatology Center — Rancho Santa Margarita, California, United States (Recruiting)
- Skin Research Institute — Coral Gables, Florida, United States (Recruiting)
- UMass Memorial Medical Center — Worcester, Massachusetts, United States (Recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
- Texas Dermatology and Laser Specialists — San Antonio, Texas, United States (Recruiting)
Study contacts
- Study coordinator: David Sweet, MD, PhD
- Email: dsweet@krystalbio.com
- Phone: 412-586-5830
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.