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
Recruiting now
Conditions studied: Recessive Dystrophic Epidermolysis Bullosa
In brief
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 facts
- Study ID
- NCT07700966
- Run by
- Abeona Therapeutics, Inc
- People needed
- 250
- Starts
- 2025-07-25
- Expected to finish
- 2045-04-01
- Last updated by the study team
- 2026-07-14
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- Inability to properly follow the protocol as determined by the Principal Investigator (PI).
Where it is running
- Lucile Packard Children's Hospital — Palo Alto, California, United States (enrolling)
- Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology — Chicago, Illinois, United States (enrolling)
- University of Texas Medical Branch — Galveston, Texas, United States (enrolling)
Full record on ClinicalTrials.gov
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