Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
Condition: Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa
MeSH terms: Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica
Intervention: Topical Beremagene Geperpavec; Placebo
Intervention description: Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein; Matching masked inactive topical gel
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: An intra-subject parallel study. Primary wounds are randomized within each subject, such that one wound receives B-VEC and the other wound receives placebo. Secondary wounds are selected to receive B-VEC only.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 31.0
Sex: All
Minimum age: 6 Months
Healthy volunteers: No
Keywords: DEB

Summary:

      To determine whether administration of topical B-VEC improves wound healing as compared to
      placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain
      safety and tolerability data.
    

Eligibility criteria:

        Inclusion Criteria:

          1. The subject or legally appointed and authorized representative must have read,
             understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved
             Informed Consent or Assent Form and must be able to and willing to follow study
             procedures and instructions.

          2. Age ≥ 6 months and older at the time of Informed Consent.

          3. Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.

          4. Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including
             COL7A1.

          5. Two (2) cutaneous wounds meeting the following criteria:

               1. Location: similar in size, located in similar anatomical regions, and have
                  similar appearance

               2. Appearance: clean with adequate granulation tissue, excellent vascularization,
                  and do not appear infected.

          6. Subjects and caregivers who, in the opinion of the Investigator, are able to
             understand the study, co-operate with the study procedures and are willing to return
             to the clinic for all the required follow-up visits.

          7. Male or Female of childbearing potential must use a reliable birth control method
             throughout the duration of the study and for three (3) months post last dose of B-VEC.

          8. Negative pregnancy test at Visit 1 (Week 1), if applicable.

        Exclusion Criteria:

          1. Medical instability limiting ability to travel to the Investigative Center.

          2. Diseases or conditions that could interfere with the assessment of safety and efficacy
             of the study treatment and compliance of the subject with study visits/procedures, as
             determined by the Investigator.

          3. Current evidence or a history of squamous cell carcinoma in the area that will undergo
             treatment.

          4. Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).

          5. Active drug or alcohol addiction as determined by the Investigator.

          6. Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).

          7. Participation in an interventional clinical trial within the past three (3) months
             (not including BVEC administration).

          8. Receipt of a skin graft in the past three (3) months.

          9. Pregnant or nursing women.