Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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|ClinicalTrials.gov Identifier: NCT04227106|
Recruitment Status : Active, not recruiting
First Posted : January 13, 2020
Last Update Posted : April 6, 2022
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa||Biological: EB-101||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)|
|Actual Study Start Date :||January 10, 2020|
|Estimated Primary Completion Date :||September 2022|
|Estimated Study Completion Date :||September 2022|
One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds
autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7
Other Name: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]
- Wound Healing [ Time Frame: 24 weeks post-treatment ]Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
- Pain Reduction [ Time Frame: 24 weeks post-treatment ]Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04227106
|United States, California|
|Redwood City, California, United States, 94063|
|United States, Massachusetts|
|University of Massachusetts Medical School|
|Worcester, Massachusetts, United States, 01605|
|Principal Investigator:||Jean Tang, MD||Stanford University|