A Phase 1/2 interventional study of Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP) in Junctional Epidermolysis Bullosa, sponsored by Holostem s.r.l.. Terminated at 1 site in Austria. Open to participants aged 6 Years to 54 Years. Per ClinicalTrials.gov, last updated 2022-03-07.
Sponsored by Holostem s.r.l. · Phase 1/2, Interventional, and Treatment
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 COL17A1 complementary DNA (cDNA) for restoration of the epidermis in patients with junctional 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 17) in patients suffering of junctional epidermolysis bullosa (JEB) with COL17A1 mutation.
This is a 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 17) will be implanted into the selected area. The specialist surgeon will either use a local or general anaesthetic for the transplant 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.
Exclusion Criteria:
Allergy, sensitivity or intolerance to drugs, excipients or other material (as per Investigator's brochure):
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. In the second surgery, genetically corrected cultured epidermal autograft (Hologene17) 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.
Other: Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP)
Surgergical procedure for transplantation under anaesthesia of genetically corrected cultured epidermal autograft (Hologene17) on blistering skin areas of JEB patients. By taking some autologous epidermal cells, a new layer of transgenic tissue is grown in the laboratory. This layer of tissue - containing genetically modified stem cells - is then implanted by a surgeon into the damaged area. The implantation can be done in one or more areas and repeated in case of failure of the first surgery.
Also known as: Transplantation of Hologene17 study product (ATMP)
Safety events (ADRs and SAEs) related to the study treatment (tolerability)
Number of patients experiencing treatment-related adverse events (TRAEs) - either to the epidermal graft and to the surgical procedures. Adverse events (serious and not serious) and adverse drug reactions (ADRs) will be collected and described
Time frame: 3 months after treatment
Safety events (ADRs and SAEs) related to the study treatment (tolerability)
Percentage of patients experiencing treatment-related adverse events (TRAEs) - either to the epidermal graft and to the surgical procedures. Adverse events (serious and not serious) and adverse drug reactions (ADRs) will be collected and described
Time frame: 12 months after treatment
Skin stability in the short term (Treatment efficacy)
The skin stability will be evaluated by means of stripping test and visual inspection of the treated areas
Time frame: 3 months after transplantation
Skin functionality in the short term (Treatment efficacy)
The skin functionality will be evaluated by means of molecular testing on skin punch biopsies taken from the transplanted areas.
Time frame: 3 months after transplantation
Skin stability in the long term (Treatment efficacy)
The skin stability will be evaluated by means of stripping test and visual inspection of the treated areas
Time frame: 12 months after transplantation
Skin functionality in the long term (Treatment efficacy)
The skin functionality will be evaluated by means of molecular testing on skin punch biopsies taken from the transplanted areas.
Time frame: 12 months after transplantation
Plan to share: No
This study is terminated, as verified in Feb 2022. You cannot join it, but the record below documents what was studied.
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Holostem s.r.l.