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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Abeona Therapeutics, Inc
Study ID
NCT07700966
Status
Recruiting
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Conditions

  • Recessive Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

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).

Study Details

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.

Key Dates

First listed
Jul 14, 2026
Start date
Jul 25, 2025
Status verified
Jul 2026
Primary completion
Apr 30, 2045
Completion
Apr 30, 2045

Study Design

Enrollment
250 participants (estimated)

Arms

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

Primary Outcome Measure

The number and incidence of treatment-related malignancies. [ Time Frame: From enrollment to 15 years post-treatment ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Lucile Packard Children's HospitalPalo AltoCalifornia94304
Thomas Buschbacher
[email protected]
650-313-0354
Joyce Teng (PRINCIPAL_INVESTIGATOR)
Children's Hospital ColoradoAuroraColorado80045
Matthew Brien
[email protected]
7207770802
Anna Bruckner (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of Chicago - Department of DermatologyChicagoIllinois60611
Alyssa Appleman
[email protected]
3125035927
Amy Paller (PRINCIPAL_INVESTIGATOR)
University of Texas Medical BranchGalvestonTexas77555
Lindsay Allen
[email protected]
4092662678
Lindy Ross (PRINCIPAL_INVESTIGATOR)

Find similar trials in Palo Alto, CA

By research site
Lucile Packard Children's Hospital· Palo Alto, CAChildren's Hospital Colorado· Aurora, COAnn & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology· Chicago, ILUniversity of Texas Medical Branch· Galveston, TX

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