A Study Assessing B-VEC Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa

Last updated: June 9, 2025
Sponsor: Krystal Biotech, Inc.
Overall Status: Active - Recruiting

Phase

3

Condition

Epidermolysis Bullosa

Treatment

Ophthalmic Beremagene Geperpavec (B-VEC)

Placebo

Clinical Study ID

NCT07016750
B-VEC-EYE-01
  • Ages > 6
  • All Genders

Study Summary

B-VEC-EYE-01 is a Phase 3 double-blind, intra-patient crossover study, to evaluate the safety and efficacy of ophthalmic Beremagene Geperpavec (B-VEC) versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. The subject and/or their parent/legal guardian must provide informed consent/assentand 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 B-VEC-EYE-NHS study.

Exclusion

Exclusion Criteria:

  1. Initiation of any new treatment regimen or change in treatment for ocular diseaseduring the run-in period except for preservative free topical antibiotics orartificial tears/lubricants associated with standard of care treatment of cornealabrasions.

  2. Treatment with an investigational agent or off-label ophthalmic use of an approvedproduct during the run-in period or planned use during the study (Exceptions may beapproved by the medical monitor on a case-by-case basis).

  3. Any condition that, in the opinion of the Investigator, would impact the completionof 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 requirementsof the study protocol in the opinion of the Investigator.

Study Design

Total Participants: 16
Treatment Group(s): 2
Primary Treatment: Ophthalmic Beremagene Geperpavec (B-VEC)
Phase: 3
Study Start date:
June 01, 2025
Estimated Completion Date:
March 31, 2026

Study Description

Subjects participating in the Sponsor's natural history study (protocol B-VEC-EYE-NHS) for at least 12 weeks and that meet all other eligibility criteria receive weekly ophthalmic administration of Investigational product (IP), B-VEC or placebo, for the first 12-week intervention period. During the second intervention period subjects will receive the alternative IP for an additional 12 weeks. IP will be administered in the home setting by a healthcare provider. 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.

Connect with a study center

  • Mission Dermatology Center

    Rancho Santa Margarita, California 92688
    United States

    Active - Recruiting

  • UMass Memorial Medical Center

    Worcester, Massachusetts 01605
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Texas Dermatology and Laser Specialists

    San Antonio, Texas 78218
    United States

    Active - Recruiting

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