Actively Recruiting
Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) to Treat Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Led by CHU de Quebec-Universite Laval · Updated on 2026-03-18
9
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new gene-modified skin graft called GMEB-SASS to help wounds heal in people with Recessive Dystrophic Epidermolysis Bullosa (RDEB). This first-in-human trial aims to assess the safety and effectiveness of this graft, which contains the patient's own living skin cells genetically modified in the lab to produce a missing protein called type VII collagen. The study includes both adults and children and is divided into two phases to gather safety data first in adults before treating pediatric patients. The GMEB-SASS graft is created by growing the patient's skin cells in the lab and using a virus-like tool to correct the genetic defect. The treatment involves two surgical steps: first, temporary allogeneic skin grafts are applied to prepare the wound bed, followed by removal and application of the GMEB-SASS graft. The graft is designed to integrate permanently with the patient's skin and covers up to 9% of the total body surface area per session, with the number of grafts based on the participant's height, weight, and age. Participants will receive wound debridement and grafting procedures and be monitored for up to 12 months. Researchers will assess safety by tracking adverse events and measure pain and itch levels using visual scales at various time points. They will also evaluate how much of the grafted area has healed and use clinical scoring tools to assess disease activity and scarring. The study involves regular follow-ups to monitor healing and safety over one year after the grafting procedure.
CONDITIONS
Brief Title
GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 18 years or older for subjects 1 to 3 in the learning phase
- Participants aged 12 years or older for subjects 4 to 6 in the learning phase
- Participants aged 7 years or older for subjects 7 to 9 in the learning phase
- Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene
- Ability to understand the study and comply with procedures, or have a caregiver who can
- Presence of one or more blistered or erosive skin areas on trunk or extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts on the day of grafting
- Ability to undergo anesthesia
You will not qualify if you...
- Medical instability limiting ability to travel to study center
- Any medical condition or illness that may affect participation or safety as judged by investigator
- Evidence of systemic infection
- Current or past non-metastatic or metastatic squamous cell carcinoma at graft site
- Clinically significant abnormal lab values or findings that may compromise safety
- History of or allergy to bovine proteins
- Active drug or alcohol addiction
- Female participants who are pregnant or breastfeeding
- Immunotherapy including oral corticosteroids over 1 mg/kg for more than one week within 2 weeks prior to intervention (topical or intranasal allowed)
- Chemotherapy within 60 days prior to intervention
- Gene therapy or products modifying collagen 7 expression within 6 months prior to intervention
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 3 to 5 days
Participants undergo wound debridement followed by the application of temporary allogeneic skin grafts to prepare the graft bed.
1 to 2 visits depending on graft preparation
Duration - Permanent graft integration expected
Participants receive the genetically modified epidermolysis bullosa self-assembled skin substitute (GMEB-SASS) graft applied to the prepared skin wound area.
1 grafting visit (in-person)
Duration - Up to 12 months
Participants are monitored for safety and efficacy, including assessments of pain, itch, graft healing, and disease activity.
Visits at baseline, week 2, and months 1, 3, 6, and 12 post intervention
Trial Site Locations
Total: 1 location
1
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G 1E8
Actively Recruiting
Research Team
L
Lucie Germain, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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