Clinical Trial: Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
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-retrovi
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 Summary:
This is a monocentric, prospective, open label, uncontrolled clinical trial, phase I/II.
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 p
Sponsor: Holostem Terapie Avanzate s.r.l.
Current Primary Outcome: Safety [ Time Frame: 3-month ]
Original Primary Outcome: Safety [ Time Frame: 3-month ]
Current Secondary Outcome: Efficacy [ Time Frame: 3- and 12-months ]
Percentage of patients with clinical success after one or more treatments with study product at 3 and 12 months follow up.
Clinically success is reached when both the following conditions are met:
o Regeneration of a clinically normal appearing skin with absence of detectable blister.
AND
o Restoration of type VII collagen expression and restoration of anchoring fibrils in the treated area.
Original Secondary Outcome: Efficacy [ Time Frame: 3- and 12-months ]
Information By: Holostem Terapie Avanzate s.r.l.
Dates:
Date Received: December 2, 2016
Date Started: January 30, 2017
Date Completion: December 2018
Last Updated: February 27, 2017
Last Verified: December 2016
