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

Condition(s) studied

Investigational drug(s) / intervention(s)

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).

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.

Eligibility

Primary outcome measure(s)

• 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

Trial sites (4)

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