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

NCT07193134 | Recruiting Phase 1 DMC

• 2 other identifiers: LOEX 020291541
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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

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

Outcome Measures

Primary Outcomes (3)

• Adverse events

  Record of Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs).

  Up to 12 months

• Pain score changes using a Visual Analogue Scale (VAS)

  Pain score of the grafted wounds for the past 7 days. Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No pain; 10 = Worst pain possible.

  Baseline, week 2, months 1, 3, 6 and 12

• Itch score changes using a Visual Analogue Scale (VAS)

  Itch score of the grafted wounds for the past 7 days (100-point visual analog scale). Horizontal line, typically 10 centimeters in length, anchored by two verbal descriptors: 0 = No itch, 100 = Worst itch Time Frame: Baseline, week 2, months 1, 3, 6 and 12

  Baseline, week 2, months 1, 3, 6 and 12 post intervention

Secondary Outcomes (3)

• Percentage of the grafted GMEB-SASS surface area that has healed

  Week 2, months 1, 3, 6 and 12 post intervention

• iscorEB questionnaire - clinician portion

  Baseline, months 3 and 12 post intervention

• Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)

  Time Frame: Baseline, months 3 and 12 post intervention

Other Outcomes (9)

• Quality of Life Epidermolysis Bullosa (QOLEB) questionnaire

  Baseline, months 1, 3, 6 and 12

• Quality-of-life EQ-5D questionnaire

  Baseline, months 1, 3, 6 and 12

• Quality-of-life iscorEB questionnaire - patient portion

  Baseline, months 1, 3, 6 and 12

Study Arms (1)

GMEB-SASS

EXPERIMENTAL

All patients will receive GMEB-SASS

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

Interventions

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

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.

GMEB-SASS

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CHU de Quebec-Universite Laval: lead

Study Sites (1)

The Hospital for Sick Children (SickKids)

Toronto, Ontario, M5G 1E8, Canada

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; 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

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lucie Germain, PhD

  CHU de Québec-Université Laval

  STUDY DIRECTOR

• Elena Pope, MD, FRCPC

  The Hospital for Sick Children (SickKids)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lucie Germain, PhD

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2025
• First Posted: September 25, 2025
• Study Start: January 7, 2026
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2035
• Last Updated: March 18, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)