Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.

NCTID	NCT02984085 (View at clinicaltrials.gov)
Description	Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying COL7A1 complementary DNA (cDNA) for restoration of the epidermis in patients with recessive dystrophic epidermolysis bullosa. The purpose of this study is to demonstrate the safety and efficacy after one or more treatments with genetically corrected cultured epidermal autograft (Hologene 7) in patients suffering of recessive dystrophic epidermolysis bullosa (RDEB) with COL7A1 mutation. (Show More)
Development Status	Inactive
Indication	Recessive Dystrophic Epidermolysis Bullosa
Disease Ontology Term	DOID:4959
Compound Name	HOLOGENE7
Compound Description	Genetically corrected cultured epidermal autograft (ATMP)
Sponsor	Holostem s.r.l.
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	3
Results Posted	Not Available

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	Epidermal cells
Delivery System	Viral transduction
Vector Type	RV
Editor Type
Dose 1	Transduced autologous skin grafts
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	Austria

Has US IND	False
FDA Designations
Recent Updates	3 patients enrolled, study terminated, to be replaced by a new study

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