NCT04227106

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Abeona Therapeutics, Inc · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).

Detailed description

Recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare, severe inherited blistering skin disease caused by the absence of a protein known as type 7 collagen (C7). There is no approved treatment for RDEB. Only supportive care is currently possible. This open-label, controlled study will evaluate the efficacy and safety of EB-101 for the treatment of large, chronic, RDEB wounds. The study intervention consists of one-time surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 10-15 participants. A single EB-101 sheet is able to provide healing to a wound area up to approximately 40cm2. Up to 6 (six) EB-101 sheets may be applied to each patient, depending on the area of existing wounds. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). Patient-reported outcomes and safety will also be collected throughout the study. The primary analysis for efficacy will be assessed when all patients reach Week 24. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 26 post-treatment. Upon completion of the study period, patients will be monitored annually as per standard of care for up to 15 years.

Conditions

• Epidermolysis Bullosa
• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	EB-101	autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7

Timeline

• Start date: 2020-01-10
• Primary completion: 2022-10-18
• Completion: 2022-10-18
• First posted: 2020-01-13
• Last updated: 2022-12-05

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study