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NCT Number: NCT07700966

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

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.

Recruiting

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Prademagene Zamikeracel

Combination Product

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).

Other names: EB-101, Pz-cel

Primary outcomes

  1. The number and incidence of treatment-related malignancies.

    Time frame: From enrollment to 15 years post-treatment

  2. The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs

    Time frame: From enrollment to 15 years post-treatment

  3. The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs.

    Time frame: From enrollment to 15 years post-treatment

  4. 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 15 years post-treatment

Other outcomes

  1. Categorical impression of change in wound healing over time via the modified Caregiver Global Impression of Change for Wound Healing (mCrGI-WH)

    Time frame: Yearly, from 1- year post-treatment to 15- years post-treatment

Study contacts

Contact information is provided by the study sponsor or research team.

Angela Iheanacho

CONTACT

[email protected]

646-813-7166

Sponsors and collaborators

Lead sponsor

Abeona Therapeutics, Inc

Industry

Registry information

Important dates

Study start
2025
Primary completion
2045
Study completion
2045
First posted
Jul 14, 2026
Registry last updated
Jul 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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