The Master Protocol includes 3 studies (Study A, Study B, and Study C) that will evaluate pz-cel and related processes in the post-marketing setting.
Study A investigates the efficacy and safety of non-conforming pz-cel in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).
Study B enables the collection of additional biopsy samples from patients receiving treatment with pz-cel.
Study C assesses the efficacy and safety of pz-cel in patient populations not represented in the VIITAL clinical trial (NCT04227106).
Willing and able to provide informed consent/assent; if under the age of 18, guardian(s) is (are) willing and able to provide consent.
Patients who are expected to receive pz-cel manufactured as intended for commercial treatment; however, the final manufactured product was non-conforming and therefore did not meet commercial release criteria.
All women of childbearing potential should discuss reproductive and breastfeeding plans and precautions with the treating physician in accordance with the considerations for special populations in the United States Prescribing Information (USPI).
Inability to adequately follow the protocol and ensure the protection of cellular sheet sites, as determined by the Investigator.
Hypersensitivity to vancomycin or amikacin.
The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as, but not limited to, active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
Evidence of systemic infection.