Clinical trial NCT07016750: A Study Assessing B-VEC Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
Official title: A Double-Blind Crossover of Ophthalmic Beremagene Geperpavec (B-VEC) and Matched Placebo, for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Dystrophic Epidermolysis Bullosa
DEB - Dystrophic Epidermolysis Bullosa
Recessive Dystrophic Epidermolysis Bullosa
Dominant Dystrophic Epidermolysis Bullosa
Keywords: Dystrophic Epidermolysis Bullosa
DEB
Corneal Abrasions
Lead sponsor: Krystal Biotech, Inc.
Sponsor class: INDUSTRY
First posted: 2025-06-12
Enrollment: 16
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

Eligibility 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 B-VEC-EYE-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.

Primary outcomes:
measure: To evaluate the safety and tolerability of ophthalmic B-VEC.
timeFrame: 24 weeks
measure: To evaluate the effect of ophthalmic B-VEC versus placebo on frequency of corneal. abrasion symptoms.
timeFrame: 24 weeks
Secondary outcomes:
measure: To evaluate the effect of ophthalmic B-VEC versus placebo on eye pain.
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Rancho Santa Margarita
country: United States
facility: Mission Dermatology Center
state: California
city: Worcester
country: United States
facility: UMass Memorial Medical Center
state: Massachusetts
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: San Antonio
country: United States
facility: Texas Dermatology and Laser Specialists
state: Texas
Study officials:
References: