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Clinical Trial Report
Gene Therapy Trial Report
Summary
Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts
NCTID
NCT04186650
(View at clinicaltrials.gov)
Description
This phase I/II 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).
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Development Status
Active
Indication
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term
DOID:4959
Compound Name
EF1a-COL7A1-SIN retroviral vector transduced autologous skin
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France
Funder Type
Other gov
Recruitment Status
Active not recruiting
Enrollment Count
3
Results Posted
Not Available
Therapy Information
Target Gene/Variant
COL7A1
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Skin graft
Drug Product Type
Autologous cells
Target Tissue/Cell
Keratinocytes
Delivery System
Viral transduction
Vector Type
RV
Editor Type
none
Dose 1
Up to 6 grafts of 50 cm^2 each
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2019-11-25
Completion Date
2027-06-09
Last Update
2025-01-29
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
France
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (GENEGRAFT)
EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa
News and Press Releases
https://cure-eb.org/research-portfolio/ebgraft/
Preclinical Publications
HEK293-based production platform for γ-retroviral (self-inactivating) vectors: application for safe and efficient transfer of COL7A1 cDNA
Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector
SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa