This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa).
The GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts. It is made in a laboratory using a small sample of the patient's own skin.
To help the patient's skin cells produce a missing protein called type VII collagen, scientists grow the patient's cells in the lab and use a virus-like tool (called a retroviral vector) to give the cells the correct instructions. This allows the cells to make the normal protein that is missing in people with RDEB.
The graft is designed to be permanent, and the goal is to improve wound healing by replacing damaged skin cells with healthy ones.
Age
Subjects 1 to 3: Eighteen (18) years old or older.
Subjects 4 to 6: Twelve (12) years old or older.
Subjects 7 to 9: Seven (7) years old or older.
Medical instability limiting the ability to travel to the investigative center.
Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment.
Evidence of systemic infection.
Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted.
Single group
Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)
DrugWound debridement will be performed, followed by the application of temporary allogeneic skin grafts for 3-5 days. The allografts will then be removed, and the GMEB-SASS grafts will be applied.
Adverse events
Record of Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs).
Pain score changes using a Visual Analogue Scale (VAS)
Pain score of the grafted wounds for the past 7 days. Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No pain; 10 = Worst pain possible.
Itch score changes using a Visual Analogue Scale (VAS)
Itch score of the grafted wounds for the past 7 days (100-point visual analog scale). Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No itch, 100 = Worst itch Time Frame: Baseline, week 2, months 1, 3, 6 and 12
Percentage of the grafted GMEB-SASS surface area that has healed
Percentage of epithelialization
iscorEB questionnaire - clinician portion
Instrument for scoring clinical severity outcomes for research of EB. iscorEB is a measurement tool for evaluating the disease severity in EB patient. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Score ranges between 0-138 for the clinician subscore.
Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)
Instrument for scoring clinical severity outcomes for research of EB. Total EBDASI activity score ranges of 0-42 (mild), 43-106 (moderate) and 107-506 (severe).
Quality of Life Epidermolysis Bullosa (QOLEB) questionnaire
Instrument for scoring quality-of-life. 17 questions covering physical, emotional, and social aspects of living with EB, with four response choices from "not at all" to "constant".
Quality-of-life EQ-5D questionnaire
Tool developed by the EuroQol Group to measure health-related quality of life. It evaluates health across five dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension has 5 levels: from no problems to extreme/unable. The tool includes a visual analogue ccale (EQ VAS): a vertical scale from 0 ("worst health imaginable") to 100 ("best health imaginable") where patients rate their overall health.
Quality-of-life iscorEB questionnaire - patient portion
Instrument for scoring clinical severity outcomes for research of EB. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. The patient portion of the iscorEB instrument, is considered a quality-of-life measure because it captures the patient's internal perceptions and experiences. Score ranges between 0-120 for the patient subscore.
Scar evaluation - Vancouver scar scale (VSS)
The Vancouver Scar Scale (VSS) is a tool used to measure scar severity across four aspects: vascularity (0-3), pliability (0-5), pigmentation (0-3), and scar height (0-4). The total score ranges from 0 (best outcome) to 15 (worst outcome).
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