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.
Single group
Non-conforming pz-cel surgical application to RDEB wounds
Biological/VaccineNon-conforming pz-cel is pz-cel intended for commercial treatment that did not meet commercial release criteria, but had no safety concerns associated with its use.
Additional biopsies
Procedure/SurgeryAdditional biopsy samples from patients who are expected to receive treatment with pz-cel in the post-marketing setting.
Pz-cel surgical application to DEB wounds
Biological/VaccineThis intervention is for patients requiring special access for application of pz-cel.
Study A - Wound Healing ≥50%
Proportion of RDEB wounds with healing ≥50% from Baseline at Week 24 (Month 6) as determined by direct Investigator assessment.
Study A - Pain Severity Scale (Wong-Baker FACES)
Pain reduction assessed by the Wong-Baker FACES Pain Rating Scale (for patients ≥6 years of age) at Week 24 (Month 6). The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Study C - Wound Healing ≥50%
Proportion of wounds with healing ≥50% from Baseline at Week 24 (Month 6) as determined by direct Investigator assessment.
Study C - Pain Severity Scale (Wong-Baker FACES)
Pain reduction assessed by Wong-Baker FACES Pain Rating Scale (for patients ≥6 years old) at Week 24 (Month 6). The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Study A - Wound Healing ≥50%
Proportion of RDEB wounds with ≥50% healing at Week 12 (Month 3) as determined by direct Investigator assessment.
Study A - Wound Healing ≥75%
Proportion of RDEB wounds with ≥75% healing at Weeks 12 (Month 3) and 24 (Month 6) as determined by direct Investigator assessment.
Study A - Wound Healing Complete
Proportion of RDEB wounds with complete healing (i.e., re-epithelialization with no drainage or erosion and presence of only minor crusting) at Weeks 12 (Month 3) and 24 (Month 6) as determined by direct Investigator assessment.
Study A - Pain Severity Scale (Wong-Baker FACES)
Pain reduction assessed by the Wong-Baker FACES Pain Rating Scale (for patients ≥6 years of age) at Week 12 (Month 3). The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Study A - The number of treatment-related cutaneous malignancies.
The number and incidence of treatment-related cutaneous malignancies.
Study A - The number of treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
The number and incidence of treatment-emergent adverse events (AEs) and serious adverse events (SAEs) related to pz-cel.
Study A - The number of positive replication-competent retrovirus (RCR) testing results
The number and incidence of positive replication-competent retrovirus (RCR) testing results required for AEs and SAEs where retroviral infection is a consideration.
Study A - The number of all treatment-emergent AEs and SAEs.
The number and incidence of all treatment-emergent AEs and SAEs.
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