Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB — GEM-3  

Recessive Dystrophic Epidermolysis Bullosa Clinical Trial

Official title: A Phase III Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously "KB103") for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

Status Completed

Clinical Trial Summary

To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.

Clinical Trial Description

Thirty-one (31) participants with DEB, aged 6 months or older at time of consent were enrolled for this Phase III study. The trial duration for each subject was about 6 months, with administration occurring once weekly. A Safety Follow-up Visit occurring 30 days from the date of final treatment with the Investigational Product also occurred. Each subject provided one pair of primary target wounds, with one wound from each pair randomized to be treated with B-VEC and the other wound with placebo. In addition to the primary target wound pair(s), additional wounds (secondary wounds) were selected to be treated with B-VEC. Throughout the study, participants complete questionnaires, had images captured of their study wounds, underwent physical exams, had vital signs and safety labs monitored. ;

Related Conditions & MeSH terms

• Dominant Dystrophic Epidermolysis Bullosa
• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Recessive Dystrophic Epidermolysis Bullosa

• NCT number: NCT04491604
• Study type: Interventional
• Source: Krystal Biotech, Inc.
• Status: Completed
• Phase: Phase 3
• Start date: August 17, 2020
• Completion date: January 14, 2022