Ignite Creation Date:
2024-05-06 @ 2:00 PM
Last Modification Date:
2024-10-26 @ 1:23 PM
Study NCT ID:
NCT04186650
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2021-10-19
First Post:
2019-11-25
Brief Title:
Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts
Sponsor:
Institut National de la Santé Et de la Recherche Médicale France
Organization:
Institut National de la Santé Et de la Recherche Médicale France
Study Overview
Official Title:
Phase III ex Vivo Gene Therapy Clinical Trial for RDEB Using Autologous Skin Equivalent Grafts Genetically Corrected With a COL7A1-encoding SIN Retroviral Vector
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2021-10
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:
EBGraft
Brief Summary:
This phase III clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa expressing residual C7 levels by genetically corrected autologous skin equivalent grafts on selected areas up to 300 cm2
Detailed Description:
Recessive Dystrophic Epidermolysis Bullosa RDEB is a severe orphan genetic disease responsible for skin and mucosal detachments due to a loss of adhesion of the epidermis to the underlying dermis The disease is caused by loss of function mutations of the COL7A1 encoding type VII collagen C7 which forms anchoring fibers which are essential structures for dermal-epidermal adherence Current treatments are only symptomatic and do not effectively treat or prevent the occurrence of cutaneous and mucosal detachments responsible for local and systemic complications that threaten the vital prognosis
EBGRAFT is a prospective open-label international monocentric phase III clinical trial It aims to treat 3 adult subjects with RDEB expressing residual C7 levels by genetically corrected autologous skin equivalent grafts
The skin equivalent consists of keratinocytes and fibroblasts from the patient genetically corrected ex vivo with a secure Self INactivating SIN retroviral vector expressing the COL7A1 cDNA under the control of the ubiquitous human promoter EF1a
Each patient will be grafted sequentially at Necker Hospital in Paris using autologous genetically corrected skin equivalents of approximately 300 cm2 up to 6 grafts of 50 cm2 each
The main objective is to evaluate the safety of autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector RV in adults with RDEB
The secondary objectives are
1 To evaluate the efficacy of transplanting autologous skin equivalent genetically corrected with RV SIN COL7A1 in adults with RDEB
2 To evaluate the immune response against recombinant type VII collagen C7
This clinical trial should evaluate whether the grafting of these genetically corrected autologous skin equivalents is well tolerated and whether they restore normal dermal-epidermal adherence of the grafted areas The proposed treatment aims to obtain a permanent correction of the grafted areas allowing skin healing and reducing pain It has the potential to reduce itching to prevent the occurrence of blisters and skin detachments reduce the risk of infections the duration and cost of care and also the risk of development of squamous cell carcinomas in the grafted areas
EBGRAFT is a prospective open-label international monocentric phase III clinical trial It aims to treat 3 adult subjects with RDEB expressing residual C7 levels by genetically corrected autologous skin equivalent grafts
The skin equivalent consists of keratinocytes and fibroblasts from the patient genetically corrected ex vivo with a secure Self INactivating SIN retroviral vector expressing the COL7A1 cDNA under the control of the ubiquitous human promoter EF1a
Each patient will be grafted sequentially at Necker Hospital in Paris using autologous genetically corrected skin equivalents of approximately 300 cm2 up to 6 grafts of 50 cm2 each
The main objective is to evaluate the safety of autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector RV in adults with RDEB
The secondary objectives are
1 To evaluate the efficacy of transplanting autologous skin equivalent genetically corrected with RV SIN COL7A1 in adults with RDEB
2 To evaluate the immune response against recombinant type VII collagen C7
This clinical trial should evaluate whether the grafting of these genetically corrected autologous skin equivalents is well tolerated and whether they restore normal dermal-epidermal adherence of the grafted areas The proposed treatment aims to obtain a permanent correction of the grafted areas allowing skin healing and reducing pain It has the potential to reduce itching to prevent the occurrence of blisters and skin detachments reduce the risk of infections the duration and cost of care and also the risk of development of squamous cell carcinomas in the grafted areas
Study Oversight
Has Oversight DMC:
None
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?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2016-002790-35 | EUDRACT_NUMBER | None | None |