Open Label Extension Protocol (Japan):Assess the Efficacy and Safety of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Japanese Patients with Dystrophic Epidermolysis Bullosa (DEB)

JRCT ID: jRCT2053230075

Registered date:21/07/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Dystrophic Epidermolysis Bullosa
• Date of first enrollment: 15/09/2023
• Target sample size: 3
• Countries of recruitment: United States,Japan
• Study type: Interventional

Intervention(s)

Participants 3 years or older will receive 1 Unit (~10^9 PFU (+- 0.5 log)) (Weekly Maximum Dose 5.0 x10^9 PFU) of topical administration of B-VEC (mixture of B-VEC and Hydroxypropyl Methylcellulose Excipient Gel). Patients greater than or equal to 2 months to less than 3 years will topically receive half of the volume (Weekly Maximum Dose 2.5 x10^9 PFU) that participants 3 years and older will receive.
PFU = plaque forming units

Outcome(s)

• Primary Outcome: To evaluate wound healing at 6 months, defined as complete closure of the Primary Wound following treatment with Beremagene Geperpavec (B-VEC).

Secondary Outcome

- To evaluate wound healing at 3 months, defined as complete closure of the Primary Wound following treatment with B-VEC.
- To evaluate safety and tolerability of B-VEC, based on the incidence, severity, and relatedness of AEs and SAEs.
- To evaluate safety and tolerability of B-VEC based on changes from baseline in clinical laboratory test results.

Key inclusion & exclusion criteria

• Age minimum: >= 2month old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Willing and able to give consent/assent
2. Clinical diagnosis of epidermolysis bullosa
3. Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1
4. Age: 2 months of age and older at the time of informed consent/assent
5. Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application.
6. At least one Primary Wound, also meeting the following criteria
- Appearance: clean with adequate granulation tissue, excellent vascularization, and do not appear infected

Exclude criteria

1. Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits or procedures, as determined by the Investigator
2. Pregnant or nursing women
3. Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the B-VEC application
4. Known allergy to any of the constituents of the product
5. Concurrent skin transplant or mesh skin grafting to the primary wound.
6. Participation in an interventional gene therapy clinical trial within the past three (3) months
7. Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
8. Participant's actively receiving chemotherapy or immunosuppressive therapy at Week 1.
9. Active drug or alcohol addiction as determined by the Investigator.

Related Information

• Primary Sponsor: Chien David
• Secondary ID(s): NCT04917874