Recruiting

pz-cel

Sponsor:

Abeona Therapeutics, Inc

Code:

NCT07700966

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Prademagene Zamikeracel

Study Details

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

Study ID

NCT07700966

Start date

Jul 25, 2025

Status verified date

Jul, 2026

Completion date

Apr, 2045

Anticipated

Primary completion date

Apr, 2045

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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).

Study Design

Enrollment

250 participants

Anticipated

Interventions and Outcome Measures

Arms

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

Interventions

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).

Primary outcome measure

  • The number and incidence of treatment-related malignancies. [ Time Frame: From enrollment to 15 years post-treatment ]
  • The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs [ Time Frame: 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. [ Time Frame: 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 [ Time Frame: From enrollment to 15 years post-treatment ]

Central Contacts and Locations

Central contacts

Angela Iheanacho

646-813-7166aiheanacho@abeonatherapeutics.com

Locations

Lucile Packard Children's Hospital

Recruiting

Palo Alto, California, United States, 94304

Contacts

Thomas Buschbacher

650-313-0354tbusch@stanford.edu

Principal Investigator:

Joyce Teng

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Matthew Brien

7207770802matthew.brien@childrenscolorado.org

Principal Investigator:

Anna Bruckner

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

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Alyssa Appleman

3125035927alyssa.appleman@northwestern.edu

Principal Investigator:

Amy Paller

University of Texas Medical Branch

Recruiting

Galveston, Texas, United States, 77555

Contacts

Lindsay Allen

4092662678listurm@utmb.edu

Principal Investigator:

Lindy Ross

More Information

Sponsor

Abeona Therapeutics, Inc

Last update posted

Jul 14, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Abeona Therapeutics, Inc on 2026-07-14.