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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:
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:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with RDEB who received pz-cel treatment in the post-marketing setting |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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| Measure | Description | Time Frame |
|---|---|---|
| Categorical impression of change in wound healing over time via the modified Caregiver Global Impression of Change for Wound Healing (mCrGI-WH) | Yearly, from 1- year post-treatment to 15- years post-treatment |
Inclusion Criteria:
Exclusion Criteria:
• Inability to properly follow the protocol as determined by the Principal Investigator (PI).
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RDEB patients who received pz-cel treatment
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Angela Iheanacho | Contact | 646-813-7166 | aiheanacho@abeonatherapeutics.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lucile Packard Children's Hospital | Recruiting | Palo Alto | California | 94304 | United States |
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|
| Children's Hospital Colorado | Recruiting | Aurora | Colorado | 80045 | United States |
|
| Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology | Recruiting | Chicago | Illinois | 60611 | United States |
|
| University of Texas Medical Branch | Recruiting | Galveston | Texas | 77555 | United States |
|
| ID | Term |
|---|---|
| D016108 | Epidermolysis Bullosa Dystrophica |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
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