Status:
NOT_YET_RECRUITING
GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
Lead Sponsor:
CHU de Quebec-Universite Laval
Conditions:
RDEB
Recessive Dystrophic Epidermolysis Bullosa
Eligibility:
All Genders
7+ years
Phase:
PHASE1
PHASE2
Brief 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...
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
- 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-feeding.
- Candidate who has received immunotherapy, including oral corticosteroids (Prednisolone \> 1 mg/kg), for more than one week, within 2 weeks prior to the study intervention (initial biopsy and GMEB-SASS grafting) (intranasal and topical preparations are permitted).
- Candidate who has received chemotherapy within 60 days prior to the study intervention (initial biopsy and GMEB-SASS grafting).
- Candidate who has received, in the last 6 months prior to the study intervention (initial biopsy and GMEB-SASS grafting), any gene therapy, chemical or biological product modifying collagen 7 expression.
Key Trial Info
Start Date :
October 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2035
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT07193134
Start Date
October 1 2025
End Date
December 1 2035
Last Update
September 25 2025
Active Locations (1)
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1
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada, M5G 1E8