A Phase 3 interventional study of EB-101 Surgical application of RDEB wounds in Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa and RDEB, sponsored by Abeona Therapeutics, Inc. Active, not recruiting at 2 sites in United States. Open to participants aged 12 Months and older. Per ClinicalTrials.gov, last updated 2025-06-27.
Sponsored by Abeona Therapeutics, Inc · Phase 3, Interventional, and Treatment
To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.
A multicenter, open-label, single-arm Phase 3b safety study of one-time surgical application of up to 12 autologous, LZRSE-Col7A1 gene-corrected keratinocyte sheets with C7 expression (EB-101) for the treatment of large, chronic DEB wounds in each of approximately 10-12 patients. Patients enrolled will also contribute biopsies to support manufacturing requirements. All patients will be followed through 24 weeks post-treatment.
Patients will be evaluated at their Screening Visit (D -60 to D -25), a phone call approximately 14 days after screening, and at Baseline (Day -1) prior to treatment on Day 0. Patients will remain in the hospital, or other suitable inpatient medical facility that will allow appropriate immobilization of treated wounds, as determined by the Investigator, for observation for up to 7 days (±2 days) following treatment. They will be evaluated by phone on Day 14; by telehealth visits on Weeks 4, 8, and 18; and by clinic visits on Weeks 12 and 24.
Must have at least one wound site that meets all of the following criteria:
Exclusion Criteria:
New or Previously Treated RDEB Patients
Biological: EB-101 Surgical application of RDEB wounds
EB-101 autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 Other Names: • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets \[LEAES\]
Also known as: pz-cel
Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.
Time frame: 6 months
Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)
The number of patients and wounds that have an infection or any related adverse event (AE).
Time frame: 6 months
Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )
The number of patients and wounds that result in hospitalization (SAE).
Time frame: 6 months
Safety Endpoint (Incidence of squamous cell carcinoma)
The incidence of squamous cell carcinoma (SCC).
Time frame: 6 months
Safety Endpoint RCR status
Replication-competent retrovirus (RCR) status.
Time frame: 6 months
Proportion of RDEB wounds with ≥50%, ≥75%, and complete
Proportion of RDEB wounds with ≥50%, ≥75%, and complete (i.e., re epithelialization with no drainage or erosion and presence of only minor crusting) healing at Weeks 12 and 24, as determined by direct Investigator assessment.
Time frame: 6 months
Extent of pain reduction assessed by the Wong-Baker FACES scale
Extent of pain reduction assessed by the Wong-Baker FACES scale (for patients ages ≥6 years old) at Weeks 8, 12, and 24.
Time frame: 6 months
Longitudinal change in scores of Worst Itch Numeric Rating Scale
Longitudinal change in scores of Worst Itch Numeric Rating Scale (WI-NRS; for patients ages ≥6 years old) scores assessed at Weeks 8, 12, and 24
Time frame: 6 months
Longitudinal change of Zarit Burden Interview Short Form
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at Weeks 12 and 24 (for all ages).
Time frame: 6 months
Change in Quality of Life in Epidermolysis Bullosa
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) scores assessed at Week 24 compared with pretreatment Baseline scores (completed by patients ≥11 years old or with guidance for patients 8 to 10 years old). If patient is \<8 years old, QOLEB should be completed by a legal guardian.
Time frame: 6 months
Change in Caregiver Global Impression of Pain
Change in Caregiver Global Impression of Pain (CrGI-Pain) scores assessed at Weeks 8, 12, and 24 compared with pretreatment Baseline (for all ages).
Time frame: 6 months
Comparison of how non-collagenous region 1 of the collagen VII molecule
Comparison of how non-collagenous region 1 of the collagen VII molecule (NC1) status relates to wound healing and safety profile (if applicable).
Time frame: 6 months
Comparison of how circulating C7 antibody status relates to wound healing and safety profile
Comparison of how circulating C7 antibody status relates to wound healing and safety profile (if applicable).
Time frame: 6 months
Plan to share: No
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This study is active, not recruiting, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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Abeona Therapeutics, Inc