RecruitingNCT07700966

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


Sponsor

Abeona Therapeutics, Inc

Enrollment

250 participants

Start Date

Jul 25, 2025

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Inclusion Criteria2

  • 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 Criteria1

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

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Interventions

COMBINATION_PRODUCTPrademagene Zamikeracel

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).


Locations(4)

Lucile Packard Children's Hospital

Palo Alto, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology

Chicago, Illinois, United States

University of Texas Medical Branch

Galveston, Texas, United States

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NCT07700966


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