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

NCT07016750 | Recruiting Phase 3 FDA Drug

• 1 other identifier: KB803-EYE-01
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 5

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

DEB - Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa

Keywords

Dystrophic Epidermolysis Bullosa; DEB; Corneal Abrasions

Outcome Measures

Primary Outcomes (2)

• To evaluate the safety and tolerability of KB803.

  Number of subjects with treatment related adverse events as assessed by CTCAE v5.

  24 weeks

• To evaluate the effect of ophthalmic KB803 on frequency of corneal abrasion symptoms.

  Average number of days per month with corneal abrasion symptoms.

  24 weeks

Secondary Outcomes (1)

• To evaluate the effect of ophthalmic KB803 on eye pain.

  24 weeks

Study Arms (2)

KB803

EXPERIMENTAL

KB803

Biological: KB803

Placebo

PLACEBO COMPARATOR

Vehicle

Drug: Placebo

Interventions

KB803 BIOLOGICAL

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

KB803

Placebo DRUG

Vehicle

Placebo

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Age 6 months or older at time of informed consent/assent.
• Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
• Meets minimum corneal abrasion symptom frequency in the NHS study.

You may not qualify if:

• 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.
• 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).
• 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.
• Women who are pregnant or nursing.
• Subject who is unwilling to comply with contraception requirements per protocol.
• Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (5)

Mission Dermatology Center

Rancho Santa Margarita, California, 92688, United States

RECRUITING

Skin Research Institute

Coral Gables, Florida, 33146, United States

RECRUITING

UMass Memorial Medical Center

Worcester, Massachusetts, 01605, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218, United States

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; Corneal Injuries

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous; Eye Injuries; Facial Injuries; Craniocerebral Trauma; Trauma, Nervous System; Nervous System Diseases; Corneal Diseases; Eye Diseases; Wounds and Injuries

Central Study Contacts

David Sweet, MD, PhD

CONTACT

412-586-5830; dsweet@krystalbio.com

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830; bagostini@krystalbio.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 27, 2025
• First Posted: June 12, 2025
• Study Start: June 20, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: March 12, 2026

Record last verified: 2026-03

Locations

US (5)