Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with recessive dystrophic epidermolysis bullosa (RDEB) - LENTICOL-F

King's College London0 个研究点目标入组 10 人开始时间: 2015年2月18日最近更新: 2018年8月20日

适应症

试验速览

暂无简介。

•1. Clinical and genetic diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations.
•2. A reduced number or morphologically abnormal anchoring fibrils confirmed by TEM.
•3. At least 5x3cm of intact skin on the trunk and/or extremities that is suitable for cell injections.
•4. Able to undergo local anaesthesia.
•5. Subjects aged = 17years and able to give informed consent prior to the first study intervention.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 10
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Subjects who received other investigational medicinal products within 6 months prior to enrolment into this study.
•2. Past medical history of biopsy proven skin malignancy.
•3. Subjects who have received 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).
•5. Subjects with BOTH:
•? - positive serum antibodies to C7 confirmed by ELISA and
•? - positive IIF with binding to the base of salt split skin.
•6. Subjects with positive results for HIV, Hepatitis B, Hepatitis C, HTLV or Syphilis.
•7. Subjects who are pregnant or of child-bearing potential who are neither abstinent nor practising 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 12 months after the cell injections.

发起方