Actively Recruiting

Phase 1
Phase 2
Age: 7Years +
All Genders
NCT07193134

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

Led by CHU de Quebec-Universite Laval · Updated on 2026-03-18

9

Participants Needed

1

Research Sites

516 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

Who Can Participate

Age: 7Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Subjects aged 18 years or older for subjects 1 to 3 in the learning phase
  • Subjects aged 12 years or older for subjects 4 to 6 in the learning phase
  • Subjects 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
  • Candidate or their parents/caregivers able to understand the study and comply with study procedures
  • On the day of grafting, one or more blistered 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
Not Eligible

You will not qualify if you...

  • Medical instability limiting the ability to travel to the investigative center
  • Any medical condition or illness that may impact study participation or safety, as judged by the investigator
  • Evidence of systemic infection
  • Current or past squamous cell carcinoma at the graft site
  • Clinically significant abnormal lab values or physical exam findings that could compromise safety
  • History of or known allergy to bovine proteins
  • Active drug or alcohol addiction
  • Female participants who are pregnant or breastfeeding
  • Use of immunotherapy including oral corticosteroids (> 1 mg/kg Prednisolone) for more than one week within 2 weeks before the study intervention (intranasal and topical allowed)
  • Chemotherapy within 60 days prior to study intervention
  • Gene therapy or collagen 7 modifying treatment within 6 months prior to study intervention

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

The Hospital for Sick Children (SickKids)

Toronto, Ontario, Canada, M5G 1E8

Actively Recruiting

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Research Team

L

Lucie Germain, PhD

CONTACT

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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