Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivale… (NCT04186650) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts
France3 participantsStarted 2020-01-10
Plain-language summary
This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300 cm2).
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
✓. Reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
✓. A reduced number of/or morphologically abnormal anchoring fibrils confirmed by TEM
✓. Detection of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot (WB) analysis
✓. Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting
✓. Ability to undergo anaesthesia for skin grafting procedures
✓. Subjects aged 18 years, willing and able to give informed consent
Exclusion criteria
✕. Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
✕. Past medical history of biopsy proven skin malignancy
✕. Immunotherapy including oral corticosteroids (Prednisolone \>1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study
✕. Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
✕
What they're measuring
1
Safety of grafting SIN RV-mediated COL7A1 gene-modified autologous skin equivalent: Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs)
✕. Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1\&2 or Syphilis serology
✕. Clinically significant medical, psychological or laboratory abnormalities limiting the ability of the subject to travel to the trial site(s) and to undergo grafting and follow-up procedures, as determined by the Investigator