Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector - EBGraft

INSERM0 个研究点目标入组 3 人开始时间: 2018年1月17日最近更新: 2019年6月30日

研究概览

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•1. = 18 year-old
•2. Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
•3. Significantly reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
•4. A reduced number of or morphologically abnormal anchoring fibrils confirmed by TEM
•5. Presence of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot analysis
•6. Presence of =100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting
•7. Ability to undergo anaesthesia for grafting procedures
•8. Subjects aged = 18years, willing and able to give informed consent
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 3
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
•2. Past medical history of biopsy proven skin malignancy
•3. 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
•4. Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
•5. Subjects with BOTH:
•positive serum antibodies to C7 confirmed by ELISA and
•positive IIF with binding to the base of salt split skin and/or
•positive Western blot
•6. Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1&2 or Syphilis serology
•7. 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
•8. Absence of adequate social support
•9. Subjects who are pregnant, breast-feeding or of child-bearing potential who are neither abstinent nor practicing an acceptable means of contraception when this is in line with the usual and preferred lifestyle of the subject, as determined by the Investigator, for the duration of the trial

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