Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds

NCT07717736 | Not Yet Recruiting Phase 4 FDA Drug

• 1 other identifier: Pz-cel-CL-402
• Study Type: interventional
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

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).

Conditions

Recessive Dystrophic Epidermolysis Bullosa (RDEB); Dystrophic Epidermolysis Bullosa (DEB); Epidermolysis Bullosa (EB)

Outcome Measures

Primary Outcomes (4)

• Study A

  Proportion of RDEB wounds with healing ≥50% from Baseline at Week 24 (Month 6) as determined by direct Investigator assessment.

  From Baseline at Week 24 (Month 6)

• Study A

  Pain reduction assessed by the Wong-Baker FACES Pain Rating Scale (for patients ≥6 years of age) at Week 24 (Month 6).

  At Week 24 (Month 6)

• Study C

  Proportion of wounds with healing ≥50% from Baseline at Week 24 (Month 6) as determined by direct Investigator assessment.

  From Baseline at Week 24 (Month 6)

• Study C

  Pain reduction assessed by Wong-Baker FACES Pain Rating Scale (for patients ≥6 years old) at Week 24 (Month 6).

  At Week 24 (Month 6)

Secondary Outcomes (10)

• Study A

  At Week 12 (Month 3)

• Study A

  At Weeks 12 (Month 3) and 24 (Month 6)

• Study A

  At Weeks 12 (Month 3) and 24 (Month 6)

• Study A

  At Week 12 (Month 3)

• Study A

  At Weeks 12 (Month 3) and 24 (Month 6)

Other Outcomes (8)

• Study A

  From enrollment to Week 24

• Study A

  From enrollment to Week 24

• Study A

  From enrollment to Week 24

Study Arms (3)

Study A

EXPERIMENTAL

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

Study B

OTHER

Tissue Collection Study for Patients Undergoing Biopsies for Treatment With Prademagene Zamikeracel (Pz-cel)

Procedure: Additional biopsies

Study C

EXPERIMENTAL

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

Interventions

Additional biopsies PROCEDURE

Additional biopsy samples from patients who are expected to receive treatment with pz-cel in the post-marketing setting.

Study B

Non-conforming pz-cel surgical application to RDEB wounds BIOLOGICAL

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.

Study A

Pz-cel surgical application to DEB wounds BIOLOGICAL

This intervention is for patients requiring special access for application of pz-cel.

Study C

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.
• Current evidence of squamous cell carcinoma (SCC) in the area that will undergo pz-cel application.
• Grade 3 clinical event or laboratory abnormality prior to pz-cel treatment, with the exception of abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch, which are expected in patients with severe DEB.
• Any other circumstance where the Investigator believes that it is not appropriate for the patient to participate in the study.
• Study B
• 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 treatment with pz-cel and are receiving a biopsy prior to pz-cel application in the post-marketing setting.
• \. Any circumstance where the Investigator believes that the patient may not be appropriate for participation in the study.
• Study C
• 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 were prescribed pz-cel for commercial treatment but require special access defined in the protocol for treatment to occur.
• All women of childbearing potential must have a negative urine pregnancy test and agree to use a reliable birth control method throughout the duration of the study.

Sponsors & Collaborators

• Abeona Therapeutics, Inc: lead

MeSH Terms

Conditions

Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases

Central Study Contacts

Angela Iheanacho

CONTACT

646-813-7166; aiheanacho@abeonatherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2026
• First Posted: July 21, 2026
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): November 1, 2031
• Study Completion (Estimated): July 1, 2032
• Last Updated: July 21, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share