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
Key facts
- Who runs it
- Abeona Therapeutics, Inc
- Where
- Aurora, Chicago, Galveston, and Palo Alto, United StatesSee the 4 sites
- Trial code
- Status
- Recruiting
- People taking part
- 250 people, planned
- Started
- 25 Jul 2025
- Expected to end
- Apr 2045
- Type of study
- Observational
- Treatments tested
- Prademagene Zamikeracel
- Record updated
- 14 July 2026
Who can take part
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
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Who can take part
- 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
Who cannot take part
- Inability to properly follow the protocol as determined by the Principal Investigator (PI)
Where in United States
- Children's Hospital ColoradoAurora, United StatesContact: Matthew BrienTaking people
- Ann & Robert H. Lurie Children's Hospital of Chicago - Department of DermatologyChicago, United StatesContact: Alyssa ApplemanTaking people
- University of Texas Medical BranchGalveston, United StatesContact: Lindsay AllenTaking people
- Lucile Packard Children's HospitalPalo Alto, United StatesContact: Thomas BuschbacherTaking people
About the trial
What the trial wants to find out
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:
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* 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.
The group in the trial
- Patients with RDEB who received pz-cel treatment in the post-marketing setting
Treatment: Prademagene Zamikeracel
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Record updated
- 14 July 2026