Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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|ClinicalTrials.gov Identifier: NCT01263379|
Recruitment Status : Active, not recruiting
First Posted : December 20, 2010
Last Update Posted : September 19, 2019
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa Dystrophica Epidermolysis Bullosa||Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets||Phase 1 Phase 2|
The research project involves gene transfer into keratinocytes, which are the majority of the cells in the outer layer of skin. In this gene transfer trial we plan to biopsy some skin tissue, grow the cells in a skin cell culture (sterile dishes with special fluid that allows cells to grow and multiply) and then infect the cells with a virus that we have genetically engineered to insert the correct type VII collagen gene. The cells should then make type VII collagen.
The process of inserting the correct type VII collagen gene into cells is called "gene transfer." The virus used is called a "retrovirus." The virus is made so that it only delivers the type VII collagen gene and it should not spread to other parts of the body. During the study we will check for growth of the virus.
After cells have received gene transfer, we will grow the cells in culture into a sheet of cells that look like a plastic film. We plan to graft the sheet to wounds. Grafting means we will take cells from the culture and stitch them to the patient's skin.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)|
|Actual Study Start Date :||October 5, 2010|
|Estimated Primary Completion Date :||December 31, 2023|
|Estimated Study Completion Date :||December 31, 2025|
Experimental: LEAES treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
This trial will create a graft, which we call "LEAES", of the patient's own skin that has been genetically engineered in our lab to express this missing protein.
Other Name: LEAES
- Percentage surface area of wound healing [ Time Frame: 3, 6 and 12 months post grafting ]Wound dimensions, including length, width, and area (in cm2), will be obtained using the Canfield system. Changes in dimensions between visits as well as changes in dimensions from baseline will be recorded. Dimensions of untreated wounded skin will be used for comparison
- Investigator's assessment of graft [ Time Frame: 3, 6 and 12 months post grafting ]The graft site will be clinically evaluated with a global score of: 1) 100% to 75% healed, 2) 74% to 50% healed, 3) 49% to 25% healed, 4) 25% to 1% healed, 5) complete graft loss, or 6) unable to determine.
- duration of type VII collagen production [ Time Frame: 12 weeks, 25 weeks, and 52 weeks post grafting ]Skin biopsies will be obtained to evaluate expression of type VII collagen, both NC1 and NC2 epitopes, using immuno-electron microscopy and immuno-fluorescent light microscopy
- presence of anchoring fibrils [ Time Frame: day 14, 4 weeks, 12 weeks, 25 weeks, and 52 weeks post grafting ]Skin biopsies will be obtained to observe physical development of the anchoring fibrils using electron microscopy
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): NCT01263379
|United States, California|
|Stanford University School of Medicine|
|Stanford, California, United States, 94305|
|Principal Investigator:||Jean Tang, MD, PhD||Stanford University|