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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| B-VEC | Experimental | Open label B-VEC topical treatment of DEB wounds. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Open Label Topical Beremagene Geperpavec (B-VEC) | Biological | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
|
| Measure | Description | Time Frame |
|---|---|---|
| To record safety outcomes of participants while on B-VEC. | 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. | Up to 112 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David Chien, MD | Senior Vice President of Clinical Development | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mission Dermatology Center | Rancho Santa Margarita | California | 92688 | United States | ||
| Stanford University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40220208 | Derived | 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. |
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| Redwood City |
| California |
| 94063 |
| United States |
| Pediatric Skin Research | Coral Gables | Florida | 33146 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Ascension Seton- Dell's Children Medical Group | Austin | Texas | 78723 | United States |
| ID | Term |
|---|---|
| D016108 | Epidermolysis Bullosa Dystrophica |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
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