Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Summary

The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).

Timeline

Start

2020-01-10

Primary completion

2022-10-18

Completion

2022-10-18

Outcome

Met primary endpoint

paper 35 (81%) of 43 treated wounds were at least 50% healed from baseline for prademagene zamikeracel compared with seven (16%) of 43 control wounds PMID 40570869 ↗

paper mean difference 67% [95% CI 50 to 89]; p<0·0001 PMID 40570869 ↗

paper The mean change from baseline to week 24 in wound pain was -3·07 with prademagene zamikeracel and -0·90 in controls PMID 40570869 ↗

paper mean pairwise difference -2·23 [-3·45 to -0·66]; p=0·0002 PMID 40570869 ↗

release “The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction” abeonatherapeutics.com ↗

Publications

• Tang JY, Marinkovich MP, Wiss K, McCarthy D, Truesdale A, Chiou AS, Eid E, McIntyre JK, Bailey I, Furukawa LK, Gorell ES, Harris N, Khosla RK, Peter Lorenz H, Lu Y, Nazaroff J, Grachev ID, Moore AJ. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. Lancet. 2025 Jul 12;406(10499):163-173. doi: 10.1016/S0140-6736(25)00778-0. Epub 2025 Jun 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	prademagene zamikeracel	Cell therapy	—	Other

Indications