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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Prademagene Zamikeracel.
- Who it may be relevant to
- Registry conditions: Recessive Dystrophic Epidermolysis Bullosa. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Interventions
- Combination product 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 measures
- 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]
Eligibility criteria
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).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 4 centers
- Lucile Packard Children's Hospital — Palo Alto
- Children's Hospital Colorado — Aurora
- Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology — Chicago
- University of Texas Medical Branch — Galveston
Identifiers
NCT: NCT07700966 · Pz-cel-RY-401