Long-Term Safety Follow-Up After pz-cel for RDEB
Official title: 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
Run by Abeona Therapeutics, Inc · NCT07700966 · Data updated 2026-11-12
This study follows people with RDEB who were treated with pz-cel to see how safe it is over up to 15 years and whether their wounds stay healed.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 250 (estimated) |
| Sponsor | Abeona Therapeutics, Inc Industry-sponsored |
| Locations | 4 sites (Palo Alto, California and 3 other locations) |
| Status | Recruiting |
| Started | July 25, 2025 |
| Main results expected | April 2045 |
| Study ends | April 2045 |
| Registry record updated | July 14, 2026 |
| Trial ID | NCT07700966 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Patients with Recessive Dystrophic Epidermolysis Bullosa who were treated with prademagene zamikeracel in the post-marketing setting and can consent or have a guardian provide consent.
✕Probably not a fit
Individuals who did not receive pz-cel or who are unable to participate in long-term follow-up may not experience direct benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is an observational registry that tracks long-term safety and durability of wound healing in patients who received prademagene zamikeracel in the post-marketing setting. Participants are followed annually for up to 15 years, primarily through remote data collection with optional in-person visits after year one. Key assessments include adverse events, serious adverse events, hospitalizations, malignancies, and potential retrovira, along with wound-healing durability. The study design does not assign treatment but monitors real-world outcomes in a multicenter, pediatric-focused setting.
Why it matters
Potential benefit: If successful, the registry could clarify long-term safety and sustained wound healing of pz-cel in RDEB patients, informing future care and monitoring.
How similar studies have gone: Related post-marketing follow-up registries have provided important safety data for gene and cell therapies, though long-term results vary by product and condition.
Who can join: the full rules
Show full inclusion / exclusion criteria
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: • Inability to properly follow the protocol as determined by the Principal Investigator (PI).
Where it's running
-
Lucile Packard Children's Hospital
Palo Alto, California, United States Recruiting -
Children's Hospital Colorado
Aurora, Colorado, United States Recruiting -
Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology
Chicago, Illinois, United States Recruiting -
University of Texas Medical Branch
Galveston, Texas, United States Recruiting
Study contacts
- Study coordinator: Angela Iheanacho
- Email: aiheanacho@abeonatherapeutics.com
- Phone: 646-813-7166
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Gene-modified autologous skin graft (GMEB-SASS) for RDEB
This trial will try a gene-modified skin graft grown from a patient's own cells to help wounds heal in people with recessive dystrophic epidermolysis bullosa (RDEB).
-
KB803 eye drops versus placebo for people with dystrophic epidermolysis bullosa
This trial tests whether KB803 eye drops, given three times a week, can reduce recurrent corneal abrasions in children and adults with dystrophic epidermolysis bullosa.
-
Using IV immunoglobulin to lower collagen VII antibodies in dystrophic epidermolysis bullosa
This trial tests whether IV immunoglobulin given to people with recessive dystrophic epidermolysis bullosa who are on VYJUVEK helps wounds heal faster and lowers collagen VII and HSV‑1 antibody levels.
-
Long-term follow-up for gene therapy in skin conditions
This study is checking the long-term safety of a gene therapy for people with a skin condition called dystrophic epidermolysis bullosa who have already received the treatment.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.