Recruiting
Phase 1
Phase 2

GMEB-SASS

Sponsor:

CHU de Quebec-Universite Laval

Code:

NCT07193134

Conditions

RDEB

Recessive Dystrophic Epidermolysis Bullosa

Epidermolysis Bullosa Dystrophica, Recessive

Eligibility Criteria

Sex: All

Age: 7+

Healthy Volunteers: Not accepted

Interventions

Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)

Study Details

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 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

RDEB

Recessive Dystrophic Epidermolysis Bullosa

Epidermolysis Bullosa Dystrophica, Recessive

Study ID

NCT07193134

Start date

Jan 7, 2026

Status verified date

Mar, 2026

Completion date

Dec, 2035

Anticipated

Primary completion date

Dec, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 7+

Healthy Volunteers: Not accepted

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-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.

Study Design

Enrollment

9 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: GMEB-SASS

All patients will receive GMEB-SASS

Interventions

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.

Primary outcome measure

  • Adverse events [ Time Frame: Up to 12 months ]
  • Pain score changes using a Visual Analogue Scale (VAS) [ Time Frame: Baseline, week 2, months 1, 3, 6 and 12 ]
  • Itch score changes using a Visual Analogue Scale (VAS) [ Time Frame: Baseline, week 2, months 1, 3, 6 and 12 post intervention ]

Central Contacts and Locations

Central contacts

Lucie Germain, PhD

418-525-4444lucie.germain@fmed.ulaval.ca

Locations

The Hospital for Sick Children (SickKids)

Recruiting

Toronto, Ontario, Canada, M5G 1E8

Contacts

Hanna Fadzeyeva

416-813-7835hanna.fadzeyeva@sickkids.ca

Pope Elena, MD, FRCPC

416-813-6883Elena.Pope@sickkids.ca

Principal Investigator:

Elena Pope, MD, FRCPC

More Information

Sponsor

CHU de Quebec-Universite Laval

Last update posted

Mar 18, 2026

Last verified

Mar, 2026

Keywords

  • Skin Abnormalities
  • Congenital Abnormalities
  • Epidermolysis Bullosa
  • Skin Diseases, Genetic
  • Genetic Diseases, Inborn
  • Collagen Diseases
  • Connective Tissue Diseases
  • Skin and Connective Tissue Diseases
  • Skin Diseases
  • Skin Diseases, Vesiculobullous
  • Epidermolysis Bullosa Dystrophica
  • Genetic therapy
  • Autologous Skin Graft
  • SIN Retroviral Vector
  • Type VII Collagen
  • COL7A1

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by CHU de Quebec-Universite Laval on 2026-03-18.