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RecruitingNCT07700966Updated Jul 14, 2026

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

An observational study in Recessive Dystrophic Epidermolysis Bullosa, sponsored by Abeona Therapeutics, Inc. Recruiting at 4 sites in United States. Per ClinicalTrials.gov, last updated 2026-07-14.

Sponsored by Abeona Therapeutics, Inc · Observational

Study type
Observational
Model
Case-only
Time perspective
Prospective
Enrollment
250
Sex
All
01

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.
02

Conditions studied

  • Recessive Dystrophic Epidermolysis Bullosa
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

RDEB patients who received pz-cel treatment

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

Exclusion Criteria:

  • Inability to properly follow the protocol as determined by the Principal Investigator (PI).
04

Study design

Observational model
Case-only
Time perspective
Prospective
Enrollment
250 participants (estimated)
Target follow-up
15 Years
Patient registry
Yes

Groups and cohorts

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

    Combination Product: Prademagene Zamikeracel

Interventions

  • Combination productPrademagene 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).

    Also known as: EB-101, Pz-cel

05

What researchers measure

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

06

Study locations

4 of 4 sites recruiting
  • Lucile Packard Children's Hospital
    Palo Alto, California 94304, United States
    • Thomas Buschbacher · Contact · tbusch@stanford.edu · 650-313-0354
    • Joyce Teng · Principal investigator
    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
    • Lindsay Allen · Contact · listurm@utmb.edu · 4092662678
    • Lindy Ross · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07700966
Lead sponsor
Abeona Therapeutics, Inc
Responsible party
Sponsor
First posted
Jul 14, 2026
Start date
Jul 25, 2025
Primary completion
Apr 2045 (estimated)
Completion
Apr 2045 (estimated)
Last update
Jul 14, 2026

Study contacts

Angela Iheanacho
Contact
aiheanacho@abeonatherapeutics.com
646-813-7166

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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