Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing E… (NCT03490331) | Clinical Trial Compass
TerminatedPhase 1/2
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17)
Stopped: No patient ongoing (none completed the study). Changes to the viral vector ongoing.
Austria1 participantsStarted 2018-03-19
Plain-language 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 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.
Who can participate
Age range6 Years – 54 Years
SexALL
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Inclusion criteria
✓. Signed and dated informed consent prior to any study-related procedures;
✓. Male and female patients between 6 years old to 54 years old;
✓. JEB molecular characterization by mutation analysis;
✓. NC16A antibody immunofluorescence or positive staining in Western Blot analysis with polyclonal antibody produced against a synthetic peptide corresponding to amino acids 131-145 of human COL17A1;
✓. Presence of chronic (persistent or recurrent for more than 3 months) large wounds (\>10 cm2) and/or persistent or recurrent erosions;
✓. A cooperative attitude to follow up the study procedures (Caregivers in case of minors).
Exclusion criteria
✕. Known or suspected intolerances against anaesthesia;
. Antibodies to type XVII collagen associated antigens demonstrated on indirect immunofluorescence;
✕. Clinical and/or laboratory signs of acute systemic infections at the time of screening. Patient can be re-screened after appropriate treatment;
✕. Severe systemic diseases (i.e. uncompensated diabetes mellitus);
✕. Female subjects: pregnant or lactating women and all women physiologically capable of becoming pregnant (i.e. women of childbearing potential) UNLESS they are willing to use one or more reliable methods of contraception with a Pearl index ≤1. Reliable contraception should be maintained throughout the study.
✕. Allergy, sensitivity or intolerance to drugs, excipients or other material (as per Investigator's brochure):