Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04227106|
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : January 13, 2020
|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 approximately 30 autologous, gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 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. Treatment effect will be measured as wound healing at 3 months, with follow up to 6 months post treatment, comparing treated versus matched untreated wound sites. Wound healing will be adjudicated by an independent committee not involved in the study conduct. 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 12. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 24 post-treatment.
Upon completion of the 6-month 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 2020|
|Estimated Study Completion Date :||March 2021|
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: 12 weeks post-treatment ]Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated intra-participant wound sites
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
|Contact: Abeona Clinical Operationsemail@example.com|
|United States, California|
|Redwood City, California, United States, 94063|
|Contact: Irene Bailey 650-721-7149 firstname.lastname@example.org|
|Principal Investigator: Jean Tang, MD|
|Sub-Investigator: Peter Marinkovich, MD|
|Principal Investigator:||Jean Tang, MD||Stanford University|