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This study examined the safety of a cell/gene therapy called gene-modified autologous fibroblasts in patients with recessive dystrophic epidermolysis bullosa. It included patients aged 17 and over with confirmed bi-allelic COL7A1 mutations. It was conducted in Phase 1 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 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 5x8cm of intact skin on the trunk and/or extremities that is suitable for cell injections. 4. Able to undergo local anaesthesia. 5. Subjects aged ≥ 17 years and able to give informed consent prior to the first study intervention. Exclusion Criteria: 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 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. 7. Subjects with positive results for HIV, Hepatitis B, Hepatitis C, HTLV or Syphilis.