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

NCT04917874 — Phase 3, Completed, 47 participants, Krystal Biotech, Inc.

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Open Label Treatment of Beremagene Geperpavec (B-VEC)

Registry	NCT04917874
Status	Completed
Phase	Phase 3
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	47 (Actual)
Sponsor	Krystal Biotech, Inc. (Industry)
Conditions	Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Interventions	Open Label Topical Beremagene Geperpavec (B-VEC) (Biological)
Dates	start 2021-05-25, primary completion 2023-07-31, completion 2023-07-31, first posted 2021-06-08, last updated 2024-04-09
Locations	6 sites in United States

-Summary

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.

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+Arms and interventions

Arm	Type	Interventions	Description
B-VEC	Experimental	Biological: Open Label Topical Beremagene Geperpavec (B-VEC)	Open label B-VEC topical treatment of DEB wounds.

Open Label Topical Beremagene Geperpavec (B-VEC) (Biological): Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein

+Outcome measures

Primary outcomes

To record safety outcomes of participants while on B-VEC. [Up to 112 weeks]: Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.

+Eligibility

Sex: All, ages: 2 Months to any

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)

+Locations (6)

Facility	City	Country
Mission Dermatology Center	Rancho Santa Margarita, California	United States
Stanford University	Redwood City, California	United States
Pediatric Skin Research	Coral Gables, Florida	United States
Northwestern University	Chicago, Illinois	United States
Cincinnati Children's Hospital Medical Center	Cincinnati, Ohio	United States
Ascension Seton- Dell's Children Medical Group	Austin, Texas	United States

+Publications

Results publication: Marinkovich MP, Paller AS, Guide SV, Gonzalez ME, Lucky AW, Bagci IS, Agostini B, Fitzgerald K, Chen S, Chen H, Conner MM, Krishnan SM. Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. Am J Clin Dermatol. 2025 Jul;26(4):623-635. doi: 10.1007/s40257-025-00942-y. Epub 2025 Apr 12. PMID 40220208

+Investigators

David Chien, MD, Study director, Senior Vice President of Clinical Development