GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment (NCT07193134) | Clinical Trial Compass
RecruitingPhase 1/2
GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
Canada9 participantsStarted 2026-01-07
Plain-language summary
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.
Who can participate
Age range
7 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Age
Learning phase:
* 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.
Other Inclusion Criteria:
* Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene.
* Candidates - or their parents/caregivers if the candidates have limited comprehension, who are able to understand the study and to comply with the study procedures.
* On the day of grafting, one or more blistered and/or erosive skin areas on the trunk and/or extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts.
* Ability to undergo anesthesia.
Exclusion Criteria:
* 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.
* Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that could compromise participant safety, as per the investigator's judgment.
* History of or known allergy to bovine proteins.
* Active drug or alcohol addiction.
* Female candidate who are pregnant or breast-fee…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse events
Timeframe: Up to 12 months
2
Pain score changes using a Visual Analogue Scale (VAS)
Timeframe: Baseline, week 2, months 1, 3, 6 and 12
3
Itch score changes using a Visual Analogue Scale (VAS)
Timeframe: Baseline, week 2, months 1, 3, 6 and 12 post intervention