VIITAL Trial: 81 Percent Healing in Large RDEB Wounds via Prademagene Zamikeracel

The study findings were published in The Lancet in July 2025.

Recessive dystrophic epidermolysis bullosa (RDEB) is a debilitating genetic disorder caused by COL7A1 mutations that deplete type VII collagen, leading to chronic wounds that are often resistant to standard clinical therapies. To bridge this therapeutic gap, Jean Y Tang of Stanford University and colleagues aimed to assess the safety and clinical efficacy of a one-time surgical application of prademagene zamikeracel in achieving definitive wound healing.

The VIITAL trial was a phase 3, intrapatient-controlled trial that studied 43 matched pairs of large, chronic wounds from 11 patients with RDEB who were randomized to compare standard care against surgically applied, autologous COL7A1 gene-modified cellular sheets. The study's co-primary endpoints measured the proportion of wounds achieving at least 50% healing and the degree of pain reduction at 24 weeks within the intention-to-treat (ITT) population for participants meeting specific collagen expression and immune criteria.

Key Clinical Findings of the Study Include:

• Superior Wound Closure Rates: The study reveals that 81% (35 of 43) of wounds treated with prademagene zamikeracel achieved at least 50% healing compared to only 16% (7 of 43) of control wounds, demonstrating a significant clinical improvement (p < 0.0001).
• Significant Reduction in Pain: The therapy resulted in a substantial mean pain reduction of -3·07 from baseline to week 24, whereas the control group experienced a much lower reduction of -0·90, representing a significant pairwise difference (p=0.0002).
• Safety and Tolerability Profile: The one-time surgical application of the gene-modified cellular sheets was well tolerated by all participants, with researchers observing no serious treatment-related adverse events throughout the study period.
• Effective Management of Large Wounds: This intervention successfully addresses the severe wound burden in RDEB by promoting durable healing in chronic wounds larger than 20 cm².

The results suggest that prademagene zamikeracel achieves at least 50% healing in 81% of treated cases. This one-time surgical application significantly reduces overall disease burden by providing both durable wound closure and substantial symptomatic relief, including a mean pain reduction of -3.07.

The study concludes, "prademagene zamikeracel represents a well-tolerated therapeutic option for clinicians to effectively facilitate durable wound closure and alleviate chronic pain, thereby significantly reducing the overall disease burden for individuals with recessive dystrophic epidermolysis bullosa."

Reference

Tang JY, Marinkovich MP, Wiss K, et al. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. The Lancet. 2025;406(10499):163-173