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

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.

Timeline

Start

2020-08-17

Primary completion

2021-10-29

Completion

2022-01-14

Outcome

Met primary endpoint

registry analysis (equivalence test); B-VEC vs Placebo; p = 0.00192; responder rate difference 45.8 (95% CI 23.6 to 68.0); McNemar NCT04491604 ↗

Publications

• Guide SV, Gonzalez ME, Bagci IS, Agostini B, Chen H, Feeney G, Steimer M, Kapadia B, Sridhar K, Quesada Sanchez L, Gonzalez F, Van Ligten M, Parry TJ, Chitra S, Kammerman LA, Krishnan S, Marinkovich MP. Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa. N Engl J Med. 2022 Dec 15;387(24):2211-2219. doi: 10.1056/NEJMoa2206663.

Drugs

Indications