Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Pz-cel Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Recessive dystrophic epidermolysis bullosa, or RDEB, is a rare connective tissue disorder. RDEB is caused by a defect in the COL7A1 gene, resulting in the inability to produce Type VII collagen, which plays an important role in anchoring the dermal and epidermal layers of the skin. Characteristics of RDEB include severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. Large chronic wounds typically do not heal spontaneously and inflict the greatest pain and clinical burden on RDEB patients.

This 3b open-label safety study includes the one-time surgical application of up to 12 autologous, gene-corrected keratinocyte sheets with C7 expression (pz-cel) for the treatment of large, chronic RDEB wounds in approximately 10-12 patients. This study seeks to enroll patients who were treated previously in a pz-cel clinical trial and seek to have additional wounds treated with pz-cel or patients who have not been treated with pz-cel.  Eligible patients will be treated and followed through 24 weeks post-treatment.

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Status

The VIITAL™ study is a Phase 3 randomized clinical trial investigating pz-cel, an engineered cell therapy for the treatment of RDEB. Large chronic wounds treated in VIITAL™ measured greater than 20 cm2 of surface area and had remained open for more than six months.

The VIITAL™ study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. pz-cel was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience.

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Status

Completed

In a randomized phase 1/2a study conducted at Stanford University, pz-cel was well-tolerated and showed evidence of multi-year wound healing and pain reduction after treatment with a mean follow-up of 5.9 years and a maximum of eight years.

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Status

Completed