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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 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 250

Last updated 2026-07-14

No results posted yet for this study

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.

Conditions

Interventions

COMBINATION_PRODUCT

Prademagene 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).

Sponsors & Collaborators

  • Abeona Therapeutics, Inc

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-07-25
Primary Completion
2045-04-30
Completion
2045-04-30
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07700966 on ClinicalTrials.gov