A Phase 1/2 interventional study of Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) in RDEB, Recessive Dystrophic Epidermolysis Bullosa and Epidermolysis Bullosa Dystrophica, Recessive, sponsored by CHU de Quebec-Universite Laval. Recruiting at 1 site in Canada. Open to participants aged 7 Years and older. Per ClinicalTrials.gov, last updated 2026-03-18.
Sponsored by CHU de Quebec-Universite Laval · Phase 1/2, Interventional, and Treatment
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.
The GMEB-SASS is a skin tissue composed of living cells genetically modified in the laboratory to express a functional form of type VII collagen. The GMEB-SASS integrates with the patient's skin once grafted. The graft is autologous and expected to be permanent.
This is a first-in-human trial aiming to explore indications of the safety and efficacy of the GMEB-SASS skin graft in healing skin wounds in RDEB patients. Adults and children are included. However, a risk mitigation measure was added in order that at least some safety data from adults be available before the pediatric population is treated. Therefore, the study design is adaptative and divided into two phases: a learning phase (phase A) and a confirmatory phase (phase B).
In the present trial, the investigators hypothesize that retroviral transfer of the COL7A1 gene, combined with the use of the self-inactivated (SIN) COL7A1 vector, will restore a functional dermo-epidermal junction in the bilayer tissue-engineered skin produced at the LOEX research center by the self-assembly method. The method for the production of substitutes is similar to the one used in the ongoing clinical trial for the treatment of burn patients (ClinicalTrials.gov Identifier: NCT02350205) using SASS, with the exception of gene modification.
An important issue in RDEB patients is that their skin is colonized by bacteria due to the continuous presence of wounds. The method used to decontaminate the graft bed is crucial to ensure proper integration of the GMEB-SASS. The proposed intervention in this trial involves two surgical steps: the use of allografts to prepare the graft bed in a first step, followed by the application of the GMEB-SASS a few days later. This approach will be applied to at least participants enrolled in Phase A of the study.
The maximum daily dose per grafting session is the number of GMEB-SASS covering a maximum of 9% of the total body surface area. The number will be calculated based on the participant's height, weight, and age.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
This study's planned enrollment of 9 is close to the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Browse Epidermolysis Bullosa Dystrophica studies →CHU de Quebec-Universite Laval is the lead sponsor of 137 studies on the registry; 27 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Learning phase:
Other Inclusion Criteria:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All patients will receive GMEB-SASS
Drug: Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)
Wound 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).
Time frame: Up to 12 months
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.
Time frame: Baseline, week 2, months 1, 3, 6 and 12
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
Time frame: Baseline, week 2, months 1, 3, 6 and 12 post intervention
Percentage of the grafted GMEB-SASS surface area that has healed
Percentage of epithelialization
Time frame: Week 2, months 1, 3, 6 and 12 post intervention
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.
Time frame: Baseline, months 3 and 12 post intervention
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).
Time frame: Time Frame: Baseline, months 3 and 12 post intervention
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".
Time frame: Baseline, months 1, 3, 6 and 12
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.
Time frame: Baseline, months 1, 3, 6 and 12
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.
Time frame: Baseline, months 1, 3, 6 and 12
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).
Time frame: months 3, 6 and 12
Participant's self-perception about the persistence of healing in the treated areas
The participant is asked about the durability of the GMEB-SASS under study compared with the skin before, choosing from three options: more durable (better), no change, or less durable (worse).
Time frame: Months 3, 6 and 12
Participant's self-perception of how easily the skin blisters in the treated areas
The participant is asked how easily the GMEB-SASS under study blister compared with the skin before, choosing from three options: more resistant to blistering (better), no change, blisters more easily (worse)
Time frame: Months 3, 6 and 12
Type VII collagen protein expression
Type VII collagen protein expression in tissue biopsies
Time frame: Baseline, months 3 and 12
Autoantibody against type VII collagen
Change in the level of autoantibody against type VII collagen in the blood
Time frame: Baseline, month 3 post intervention if signs of rejection, month 12 for others
Economic impact evaluation
Economic impact evaluation questionnaire: tool to measure direct and indirect costs of a health condition for patients and their families.
Time frame: Baseline, months 1, 6, 12
Plan to share: No
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Started Jan 2026 (actual). Primary completion is estimated for Dec 2030, 59 months after the start.
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