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

Plain-language summary

This observational registry aims to evaluate long-term safety and wound healing durability for up to 15 years in people with recessive dystrophic epidermolysis bullosa (RDEB) who were treated with prademagene zamikeracel (EB-101, pz-cel) in the post-marketing setting. It also records the occurrence of any malignancy after treatment, regardless of type or location. Participants are followed annually, mostly through remote data collection; eligibility requires the ability to give consent or assent, with guardian consent for those under 18, and prior pz-cel treatment in the post-marketing setting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Abeona Therapeutics, Inc

Intervention

Prademagene Zamikeracel (EB-101, Pz-cel)

Start date

7/25/2025

Estimated completion

4/1/2045

Countries

United States

Related genes

Locations

• Palo Alto, United States
• Aurora, United States
• Chicago, United States
• Galveston, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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).