Recruiting
Phase 3

KB803

Sponsor:

Krystal Biotech, Inc.

Code:

NCT07016750

Conditions

Dystrophic Epidermolysis Bullosa

DEB - Dystrophic Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa

Dominant Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

KB803

Placebo

Study Details

Brief 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).

Conditions

Dystrophic Epidermolysis Bullosa

DEB - Dystrophic Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa

Dominant Dystrophic Epidermolysis Bullosa

Study ID

NCT07016750

Start date

Jun 20, 2025

Status verified date

Mar, 2026

Completion date

Dec, 2026

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

16 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: KB803

KB803

placebo comparator: Placebo

Vehicle

Interventions

KB803

Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein

Placebo

Vehicle

Primary outcome measure

  • To evaluate the safety and tolerability of KB803. [ Time Frame: 24 weeks ]
  • To evaluate the effect of ophthalmic KB803 on frequency of corneal abrasion symptoms. [ Time Frame: 24 weeks ]

Central Contacts and Locations

Central contacts

Locations

Mission Dermatology Center

Recruiting

Rancho Santa Margarita, California, United States, 92688

Contacts

Skin Research Institute

Recruiting

Coral Gables, Florida, United States, 33146

Contacts

UMass Memorial Medical Center

Recruiting

Worcester, Massachusetts, United States, 01605

Contacts

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Texas Dermatology and Laser Specialists

Recruiting

San Antonio, Texas, United States, 78218

Contacts

More Information

Sponsor

Krystal Biotech, Inc.

Last update posted

Mar 12, 2026

Last verified

Mar, 2026

Keywords

  • Dystrophic Epidermolysis Bullosa
  • DEB
  • Corneal Abrasions

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Krystal Biotech, Inc. on 2026-03-12.