Ignite Creation Date:
2025-12-25 @ 2:34 AM
Ignite Modification Date:
2025-12-26 @ 1:11 AM
Study NCT ID:
NCT07193134
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-09-25
First Post:
2025-09-16
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
Sponsor:
CHU de Quebec-Universite Laval
Organization:
Study Overview
Official Title:
Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) to Treat Patients Suffering From Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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.
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 grafted. The graft is autologous and expected to be permanent.
This is a first-in-human trial aiming to explore indications of the safety and efficacy of the GMEB-SASS skin graft in healing skin wounds in RDEB patients. Adults and children are included. However, a risk mitigation measure was added in order that at least some safety data from adults be available before the pediatric population is treated. Therefore, the study design is adaptative and divided into two phases: a learning phase (phase A) and a confirmatory phase (phase B).
In the present trial, the investigators hypothesize that retroviral transfer of the COL7A1 gene, combined with the use of the self-inactivated (SIN) COL7A1 vector, will restore a functional dermo-epidermal junction in the bilayer tissue-engineered skin produced at the LOEX research center by the self-assembly method. The method for the production of substitutes is similar to the one used in the ongoing clinical trial for the treatment of burn patients (ClinicalTrials.gov Identifier: NCT02350205) using SASS, with the exception of gene modification.
An important issue in RDEB patients is that their skin is colonized by bacteria due to the continuous presence of wounds. The method used to decontaminate the graft bed is crucial to ensure proper integration of the GMEB-SASS. The proposed intervention in this trial involves two surgical steps: the use of allografts to prepare the graft bed in a first step, followed by the application of the GMEB-SASS a few days later. This approach will be applied to at least participants enrolled in Phase A of the study.
The maximum daily dose per grafting session is the number of GMEB-SASS covering a maximum of 9% of the total body surface area. The number will be calculated based on the participant's height, weight, and age.
This is a first-in-human trial aiming to explore indications of the safety and efficacy of the GMEB-SASS skin graft in healing skin wounds in RDEB patients. Adults and children are included. However, a risk mitigation measure was added in order that at least some safety data from adults be available before the pediatric population is treated. Therefore, the study design is adaptative and divided into two phases: a learning phase (phase A) and a confirmatory phase (phase B).
In the present trial, the investigators hypothesize that retroviral transfer of the COL7A1 gene, combined with the use of the self-inactivated (SIN) COL7A1 vector, will restore a functional dermo-epidermal junction in the bilayer tissue-engineered skin produced at the LOEX research center by the self-assembly method. The method for the production of substitutes is similar to the one used in the ongoing clinical trial for the treatment of burn patients (ClinicalTrials.gov Identifier: NCT02350205) using SASS, with the exception of gene modification.
An important issue in RDEB patients is that their skin is colonized by bacteria due to the continuous presence of wounds. The method used to decontaminate the graft bed is crucial to ensure proper integration of the GMEB-SASS. The proposed intervention in this trial involves two surgical steps: the use of allografts to prepare the graft bed in a first step, followed by the application of the GMEB-SASS a few days later. This approach will be applied to at least participants enrolled in Phase A of the study.
The maximum daily dose per grafting session is the number of GMEB-SASS covering a maximum of 9% of the total body surface area. The number will be calculated based on the participant's height, weight, and age.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 291541 | OTHER | Health Canada | View |