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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

Recruitingn/aAges any - anyAllAbeona Therapeutics, Inc
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In plain words

What the trial is, and whether it could suit you

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), Serio

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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: โ€ข Inability to properly follow the protocol as determined by the Principal Investigator (PI).
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What it tests

  • Prademagene Zamikeracel
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Where

4 sites
Lucile Packard Children's Hospital
Palo Alto, United States
Children's Hospital Colorado
Aurora, United States
Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology
Chicago, United States
University of Texas Medical Branch
Galveston, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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