A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

Krystal Biotech

Status and phase: Completed; Phase 3

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

Treatments: Biological: Open Label Topical Beremagene Geperpavec (B-VEC)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04917874

B-VEC-EX-02

Details and patient eligibility

About

This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.

Enrollment: 47 patients

Sex: All

Ages: 2+ months old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Willing and able to give consent/assent
• Clinical diagnosis of epidermolysis bullosa
• Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
• Age: 2 months of age and older at the time of informed consent/assent
• 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

Exclusion criteria

• Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
• Pregnant or nursing women
• Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
• Known allergy to any of the constituents of the product
• Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
• Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

47 participants in 1 patient group

B-VEC

Experimental group

Description:

Open label B-VEC topical treatment of DEB wounds.

Treatment:

Biological: Open Label Topical Beremagene Geperpavec (B-VEC)