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).
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
This study's planned enrollment of 100 is above the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Browse Epidermolysis Bullosa Dystrophica studies →Abeona Therapeutics, Inc is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
Compared within Epidermolysis Bullosa Dystrophica, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Epidermolysis Bullosa. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Start is estimated for Nov 2026; primary completion for Nov 2031, 60 months later.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Epidermolysis Bullosa Dystrophica →
Abeona Therapeutics, Inc