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 FDA Drug FDA Device

• 1 other identifier: Pz-cel-RY-401
• Study Type: observational
• Target: 250
• Locations: 1 country
• Sites: 4

Brief 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

Recessive Dystrophic Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (4)

• 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 (1)

• 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

Study Arms (1)

Patients with RDEB who received pz-cel treatment in the post-marketing setting

Combination Product: Prademagene Zamikeracel

Interventions

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).

Also known as: EB-101, Pz-cel

Patients with RDEB who received pz-cel treatment in the post-marketing setting

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

RDEB patients who received pz-cel treatment

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Abeona Therapeutics, Inc: lead

Study Sites (4)

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

University of Texas Medical Branch

Galveston, Texas, 77555, United States

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Central Study Contacts

Angela Iheanacho

CONTACT

646-813-7166; aiheanacho@abeonatherapeutics.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 15 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2026
• First Posted: July 14, 2026
• Study Start: July 25, 2025
• Primary Completion (Estimated): April 1, 2045
• Study Completion (Estimated): April 1, 2045
• Last Updated: July 14, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)