An observational study in RDEB, sponsored by Abeona Therapeutics, Inc. Enrolling by invitation at 2 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-07-17.
Sponsored by Abeona Therapeutics, Inc · Observational
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.
Patient with previous EB-101 treatment
Exclusion Criteria:
This study will follow one group of participants who were enrolled in a previous EB-101 study.
Biological: EB-101
This is non-interventional and follows patients treated with EB-101
Also known as: pz-cel
Wound Closure
Proportion of RDEB wounds with healing ≥50%, ≥75%, and 100% at all clinic visits up to Month 54 as determined by direct investigator assessment.
Time frame: 5 years
Pain Severity Scale (Wong-Baker FACES)
Change in pain assessed by Wong-Baker FACES scale at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Time frame: 5 years
Itch Severity Scale
Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Time frame: 5 years
Zarit Burden Interview Scale
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 4, with the higher scores being the worse outcome.
Time frame: 5 years
Quality of Life in Epidermolysis Bullosa
Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54.
Time frame: 5 years
Caregiver Global Impression of Change for Pain Scale
Caregiver Global Impression of Change for Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54. The minimum score value is 1 and the maximum score value is 7, with the higher scores being the worse outcome.
Time frame: 5 years
Wound Infection and Adverse Events
The number of participants and wounds that have an infection or any related adverse event.
Time frame: 5 years
The number of treatment-emergent adverse events.
The number of treatment-emergent adverse events.
Time frame: 5 years
Serious Adverse Events
The number of participants and wounds that result in hospitalization (serious adverse event \[SAE\]).
Time frame: 5 years
Incidence of squamous cell carcinoma.
Incidence of squamous cell carcinoma.
Time frame: 5 years
Replication-competent retrovirus (RCR) status
Replication-competent retrovirus (RCR) status
Time frame: 5 years
Plan to share: Undecided
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Abeona Therapeutics, Inc