NCT05725018

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Abeona Therapeutics, Inc · Industry
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Epidermolysis Bullosa
• Recessive Dystrophic Epidermolysis Bullosa
• RDEB

Interventions

Type	Name	Description
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 \[LEAES\]

Timeline

• Start date: 2023-04-02
• Primary completion: 2025-09-30
• Completion: 2025-09-30
• First posted: 2023-02-13
• Last updated: 2025-06-27

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study