A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting

NCT07700966 — Recruiting, 250 participants, Abeona Therapeutics, Inc

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Registry	NCT07700966
Status	Recruiting
Study type	Observational
Enrollment	250 (Estimated)
Sponsor	Abeona Therapeutics, Inc (Industry)
Conditions	Recessive Dystrophic Epidermolysis Bullosa
Interventions	Prademagene Zamikeracel (Combination product)
Dates	start 2025-07-25, primary completion 2045-04-01 (estimated), completion 2045-04-01 (estimated), first posted 2026-07-14, last updated 2026-07-14
Locations	4 sites in United States

-Summary

The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are:

* The long-term safety profile of pz-cel.
* The wound healing durability of pz-cel.
* The occurrence of all malignancies after treatment with pz-cel, regardless of type or location, in individuals who received pz-cel treatment for RDEB in the post-marketing setting.

Participants will then be followed annually up to 15 years post-treatment. Assessments will occur via remote data collection. If the participants provider determines an office visit is necessary after the first year, some of this data can be collected in person as well. Annual assessments will include, but are not limited to:

* Review of Adverse Events (AEs), Serious Adverse Events (SAEs), hospitalizations, and any evidence of malignancies and potential retroviral infection
* Wound-healing durability data collection via the modified Caregiver Global Impression of Change for Wound Healing
* Any changes in concomitant medications and procedures.

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+Arms and interventions

Arm	Type	Interventions	Description
Patients with RDEB who received pz-cel treatment in the post-marketing setting		Combination Product: Prademagene Zamikeracel

Prademagene Zamikeracel (Combination product), also known as EB-101, Pz-cel: ZEVASKYN® (prademagene zamikeracel) is a type of gene therapy made from your own skin cells that is surgically applied to treat wounds in both children and adults who have recessive dystrophic epidermolysis bullosa (RDEB).

+Outcome measures

Primary outcomes

The number and incidence of treatment-related malignancies. [From enrollment to 15 years post-treatment]
The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs [From enrollment to 15 years post-treatment]
The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs. [From enrollment to 15 years post-treatment]
The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration [From enrollment to 15 years post-treatment]

Other outcomes

Categorical impression of change in wound healing over time via the modified Caregiver Global Impression of Change for Wound Healing (mCrGI-WH) [Yearly, from 1- year post-treatment to 15- years post-treatment]

+Eligibility

Sex: All

Inclusion Criteria:

* Willing and able to give consent/assent; if under the age of 18, guardian(s) is(are) willing and able to give consent.
* Patients who received treatment with prademagene zamikeracel in the post-marketing setting.

Exclusion Criteria:

• Inability to properly follow the protocol as determined by the Principal Investigator (PI).

+Locations (4)

Facility	City	Country	Status
Lucile Packard Children's Hospital	Palo Alto, California	United States	Recruiting
Children's Hospital Colorado	Aurora, Colorado	United States	Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology	Chicago, Illinois	United States	Recruiting
University of Texas Medical Branch	Galveston, Texas	United States	Recruiting