A Phase 4 interventional study of Non-conforming pz-cel surgical application to RDEB wounds and Additional biopsies in Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Dystrophic Epidermolysis Bullosa (DEB), sponsored by Abeona Therapeutics, Inc. Not yet recruiting. Per ClinicalTrials.gov, last updated 2026-08-06.
Sponsored by Abeona Therapeutics, Inc · Phase 4, Interventional, and Treatment
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).
The Master Protocol includes 3 studies (Study A, Study B, and Study C) that will evaluate pz-cel (LZRSE-COL7A1 gene-corrected cellular sheets with type VII collagen [C7] expression) and related processes in the post-marketing setting.
Pz-cel is a genetically engineered autologous cell therapy indicated for the treatment of wounds associated with RDEB. Pz-cel is composed of autologous keratinocytes from patients with mutations in the collagen type VII alpha 1 chain (COL7A1) gene, which have been transduced ex vivo with the Moloney leukemia virus-derived retroviral vector (LZRSE)-COL7A1. The gene-corrected cellular sheets express functional C7.
The pz-cel sheets are a one-time surgical application to debrided and cauterized wound beds of the corresponding patients with DEB. The COL7A1 transgene integrates into the host cell genome, resulting in durable expression and secretion of collagen protein, which addresses the underlying mechanism of the disease.
Study A is an open-label, non-randomized study in patients who are expected to receive gene-corrected cellular sheets (pz-cel) for the treatment of RDEB wound sites in the post-marketing setting and whose manufactured patient-specific batch of pz-cel intended for commercial treatment did not meet commercial release criteria, but had no safety concerns associated with its use. This study will allow surgical application of such non-conforming pz-cel when the benefit of application outweighs the risks. All patients will be followed through 24 weeks after treatment and may be eligible for further follow-up after Week 24. Patients will be evaluated at their Screening visit (30 days before surgery), at Day 0 (Surgery day), by phone on Week 4 (Month 1) and by Telehealth or Clinic Visit at Week 12 (Month 3) and Week 24 (Month 6).
Study B is a study to collect additional biopsy samples from patients who are expected to receive treatment with pz-cel in the post-marketing setting. The additional biopsy samples are being collected to support pz-cel product optimization. Patients receiving treatment with pz-cel can consent to have 2 additional 8-mm biopsies for use in the pz-cel assay and process development as well as in the optimization of the pz-cel manufacturing processes. The study will consist of Screening (in-person or via phone and eConsent), a pre-surgery biopsy and an End of Study Phone call 10-14 days after biopsy.
Study C is an open-label, non-randomized study in patients who are prescribed gene-corrected cellular sheets (pz-cel) in the post-marketing setting and who are part of a patient population not represented in the VIITAL Clinical Trial (NCT04227106). This study will enable patients with wounds that could benefit from treatment with pz-cel, especially those requiring special access for application of pz-cel, to receive pz-cel. All patients will be followed through 24 weeks after treatment and may be eligible for further follow-up after Week 24. Patients will be evaluated at their Screening visit (25-60 days before surgery), at Day -1 (one day prior to surgery), at Day 0 (Surgery Day), by phone on Week 4 (Month 1) and by Telehealth or Clinic Visit at Week 12 (Month 3) and Week 24 (Month 6).
Study A
Inclusion Criteria
Exclusion Criteria
Study B
Inclusion Criteria
Exclusion Criteria
1. Any circumstance where the Investigator believes that the patient may not be appropriate for participation in the study.
Study C
Inclusion Criteria:
Exclusion Criteria:
Clinical Evaluation of Prademagene Zamikeracel (Pz-cel) Treatment in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Who Were Prescribed Pz-cel and Received Non-conforming Pz-cel in the Post-marketing Setting
Biological: Non-conforming pz-cel surgical application to RDEB wounds
Tissue Collection Study for Patients Undergoing Biopsies for Treatment With Prademagene Zamikeracel (Pz-cel)
Procedure: Additional biopsies
Post-Marketing Pz-cel Access Study in Dystrophic Epidermolysis Bullosa (DEB) Evaluating Patient Populations Not Represented in the VIITAL Clinical Trial (NCT04227106)
Biological: Pz-cel surgical application to DEB wounds
Non-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 biopsy samples from patients who are expected to receive treatment with pz-cel in the post-marketing setting.
This 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.
Time frame: From Baseline at Week 24 (Month 6)
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.
Time frame: At Week 24 (Month 6)
Study C - Wound Healing ≥50%
Proportion of wounds with healing ≥50% from Baseline at Week 24 (Month 6) as determined by direct Investigator assessment.
Time frame: From Baseline at Week 24 (Month 6)
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.
Time frame: At Week 24 (Month 6)
Study A - Wound Healing ≥50%
Proportion of RDEB wounds with ≥50% healing at Week 12 (Month 3) as determined by direct Investigator assessment.
Time frame: At Week 12 (Month 3)
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.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
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.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
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.
Time frame: At Week 12 (Month 3)
Study A - Worst Itch Severity Scale
Change in scores of Worst Itch Numeric Rating Scale (WI-NRS; for patients ≥6 years of age) assessed at Weeks 12 (Month 3) and 24 (Month 6). The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
Study C - Wound Healing ≥50%
Proportion of DEB wounds with ≥50% healing at Week 12 (Month 3) as determined by direct Investigator assessment.
Time frame: At Week 12 (Month 3)
Study C - Wound Healing ≥75%
Proportion of DEB wounds with ≥75% healing at Weeks 12 (Month 3) and 24 (Month 6) as determined by direct Investigator assessment.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
Study C - Wound Healing Complete
Proportion of DEB 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.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
Study C - 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.
Time frame: At Week 12 (Month 3)
Study C - Worst Itch Severity Scale
Change in scores of WI-NRS (for patients ≥6 years of age) assessed at Weeks 12 (Month 3) and 24 (Month 6). The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Time frame: At Weeks 12 (Month 3) and 24 (Month 6)
Study A - The number of treatment-related cutaneous malignancies.
The number and incidence of treatment-related cutaneous malignancies.
Time frame: From enrollment to Week 24
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.
Time frame: From enrollment to Week 24
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.
Time frame: From enrollment to Week 24
Study A - The number of all treatment-emergent AEs and SAEs.
The number and incidence of all treatment-emergent AEs and SAEs.
Time frame: From enrollment to Week 24
Study C - The number of treatment-related cutaneous malignancies.
The number and incidence of treatment-related cutaneous malignancies.
Time frame: From enrollment to Week 24
Study C - The number of treatment-emergent AEs and SAEs
The number and incidence of treatment-emergent AEs and SAEs related to pz-cel.
Time frame: From enrollment to Week 24
Study C - The number of positive RCR testing results required for AEs and SAEs
The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration.
Time frame: From enrollment to Week 24
Study C - The number of all treatment-emergent AEs and SAEs.
The number and incidence of all treatment-emergent AEs and SAEs.
Time frame: From enrollment to Week 24
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Abeona Therapeutics, Inc