Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa
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Le résumé fourni par la source
BACKGROUND: Patients with dystrophic epidermolysis bullosa have pathogenic variants in COL7A1, leading to skin fragility. Beremagene geperpavec-svdt (B-VEC) is a modified, herpes simplex virus type 1-based gene therapy vector that topically delivers COL7A1 to dystrophic epidermolysis bullosa wounds. In a phase III study, B-VEC significantly improved wound healing at 3 and 6 months compared with placebo. OBJECTIVE: We aimed to evaluate the safety and tolerability of B-VEC beyond 6 months in patients with dystrophic epidermolysis bullosa. METHODS: An open-label extension study was conducted with 47 subjects (24 rollover from phase III; 23 treatment naïve) receiving B-VEC weekly to target wound areas for up to 112 weeks (median 81 weeks). Safety was assessed by adverse events. Treatment satisfaction and quality of life were assessed with patient-reported outcomes as exploratory measures of efficacy. Selected wounds from phase III rollover subjects were assessed for closure. RESULTS: Thirty-five subjects (74.5%) reported one or more adverse events; most were mild or moderate in severity. Fourteen subjects experienced 17 serious adverse events and ten experienced 14 severe adverse events; none was considered treatment related. No adverse events led to treatment or study discontinuation. Patient-reported outcomes indicated high levels of treatment satisfaction, but were inconclusive with regard to quality of life. Among rollover subjects, wounds that received B-VEC during phase III maintained high closure rates during the open-label extension (range 61.1-89.5%, assessed baseline to month 12). LIMITATIONS: This was an open-label design, with a variable follow-up. CONCLUSIONS: Patients undergoing extended B-VEC treatment maintained high satisfaction and continued to respond to treatment with no new safety signals detected in the open-label extension study, supporting the continuous use of B-VEC. CLINICAL TRIAL REGISTRATION: NCT04917874 (date of trial registration: 8 June, 2021).
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa
- Date Crossref
- 12/04/2025
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Veterans Health Administration pays non établi dans la noticeOrganisme public
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Stanford University Department of Dermatology pays non établi dans la noticeUniversité ou école supérieure
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Northwestern University Departments of Dermatology and Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Children's Hospital of Orange County pays non établi dans la noticeÉtablissement de santé
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Cincinnati Children's Hospital Medical Center pays non établi dans la noticeÉtablissement de santé
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Veterans Affairs Medical Center pays non établi dans la noticeÉtablissement de santé
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University of California Irvine Department of Dermatology pays non établi dans la noticeUniversité ou école supérieure
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Pediatric Skin Research pays non établi dans la noticeInstitution
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University of Cincinnati College of Medicine Cincinnati Children's Hospital Medical Center pays non établi dans la noticeUniversité ou école supérieure
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Inc. Krystal Biotech pays non établi dans la noticeEntreprise
Veterans Health Administration, Department of Dermatology — Stanford University et Departments of Dermatology and Pediatrics — Northwestern University, avec 7 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.