Ignite Creation Date:
2024-05-06 @ 9:28 AM
Last Modification Date:
2024-10-26 @ 12:14 PM
Study NCT ID:
NCT02984085
Status:
TERMINATED
Last Update Posted:
2022-02-16
First Post:
2016-12-02
Brief Title:
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB
Sponsor:
Holostem Terapie Avanzate srl
Organization:
Holostem Terapie Avanzate srl
Study Overview
Official Title:
Prospective Open-label Uncontrolled Clinical Trial to Assess the Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified With a Gamma-retroviral rv Vector Carrying COL7A1 cDNA for Restoration of Epidermis in Patients With Recessive Dystrophic Epidermolysis Bullosa
Status:
TERMINATED
Status Verified Date:
2022-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Replaced by new study in progress
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HOLOGENE7
Brief Summary:
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
Detailed Description:
This is a monocentric prospective open label uncontrolled clinical trial phase III
Patients will be screened according to the Study Inclusion and Exclusion criteria and will be candidate for the treatment if all inclusion and none of the exclusion criteria are met
After confirmation of eligibility patients will undergo biopsy for the collection of the autologous epidermal cells to be used to produce the tissue for the treatment In case all criteria are met the transplantation of the new cultured transgenic epidermis will be planned according to the procedures and the need of the patient
The study treatment consists of a surgical intervention for new restored stem cells implantation
The surgery will be carried out in 2 stages the first aims at taking biopsy to isolate epidermal cells including stem cells The biopsy will be processed in a laboratory of a regenerative medicine manufacturing site where they will be corrected expanded and prepared as final sheets to be implanted Therefore the patient can have his second intervention In this second surgery genetically corrected cultured epidermal autograft Hologene 7 will be implanted into the selected area The specialist surgeon will either use a local or general anaesthetic for the implant operation The treated area will be immobilized for some days after this operation Antibiotics and anti-inflammatory drugs will be administered if necessary to prevent infections and to minimise swelling
Three months after the transplantation primary endpoint will be evaluated by the Investigator The study completion will be reached when 1 year secondary endpoint of follow-up after the last transplant in the last patient will be accomplished
The end of the trial is defined as the last visit of the last patient after the last treatment if any
Patients will be screened according to the Study Inclusion and Exclusion criteria and will be candidate for the treatment if all inclusion and none of the exclusion criteria are met
After confirmation of eligibility patients will undergo biopsy for the collection of the autologous epidermal cells to be used to produce the tissue for the treatment In case all criteria are met the transplantation of the new cultured transgenic epidermis will be planned according to the procedures and the need of the patient
The study treatment consists of a surgical intervention for new restored stem cells implantation
The surgery will be carried out in 2 stages the first aims at taking biopsy to isolate epidermal cells including stem cells The biopsy will be processed in a laboratory of a regenerative medicine manufacturing site where they will be corrected expanded and prepared as final sheets to be implanted Therefore the patient can have his second intervention In this second surgery genetically corrected cultured epidermal autograft Hologene 7 will be implanted into the selected area The specialist surgeon will either use a local or general anaesthetic for the implant operation The treated area will be immobilized for some days after this operation Antibiotics and anti-inflammatory drugs will be administered if necessary to prevent infections and to minimise swelling
Three months after the transplantation primary endpoint will be evaluated by the Investigator The study completion will be reached when 1 year secondary endpoint of follow-up after the last transplant in the last patient will be accomplished
The end of the trial is defined as the last visit of the last patient after the last treatment if any
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