{ "data": { "asOf": "2026-10-30T22:26:48.426Z", "dedupMethod": "exact cross-registry identifier matching; probabilistic = review only", "byRegistry": { "clinicaltrials_gov": 1 }, "canonicalTrial": { "canonicalId": "NCT04186650", "title": "Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts", "scientificTitle": "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", "status": "active", "studyType": "interventional", "phase": [ "PHASE1", "PHASE2" ], "conditions": [ "Epidermolysis Bullosa Dystrophica, Recessive" ], "interventions": [ { "name": "COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin", "type": "BIOLOGICAL", "description": "Graft of SIN RV-mediated COL7A1 gene-modified autologous skin equivalent" } ], "sponsor": { "name": "Institut National de la Santé Et de la Recherche Médicale, France", "type": "OTHER_GOV" }, "locations": [ { "city": "Paris", "country": "France", "facility": "Institut Imagine Necker Hospital" } ], "outcomes": { "other": [], "primary": [ "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)" ], "secondary": [ "Change in C7 protein expression", "Change in anchoring fibrils number", "Change in scar quality: Vancouver Scar Scale (VSS)", "Changes in blister number over the grafted skin", "Changes in clinical appearance of grafted skin", "Changes in pruritus of grafted skin", "Change in Quality of life: QOLEB questionnaire (Quality of Life for Epidermolysis Bullosa)", "Change in Birmingham Epidermolysis Score (BEBS)", "Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Activity", "Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Damage", "Change in instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB).", "Evaluation of the humoral immune response against recombinant C7", "Evaluation of the cytotoxic immune response against recombinant C7" ] }, "eligibility": { "sex": "ALL", "criteria": "Inclusion Criteria:\n\n1. Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations\n2. Reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)\n3. A reduced number of/or morphologically abnormal anchoring fibrils confirmed by TEM\n4. Detection of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot (WB) analysis\n5. Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting\n6. Ability to undergo anaesthesia for skin grafting procedures\n7. Subjects aged 18 years, willing and able to give informed consent\n\nExclusion Criteria:\n\n1. Recipients of other investigational medicinal products within 6 months prior to enrolment into this study\n2. Past medical history of biopsy proven skin malignancy\n3. 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\n4. Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin\n5. Subjects with BOTH:\n\n * positive serum antibodies to C7 confirmed by ELISA and\n * positive IIF with binding to the base of salt split skin and/or\n * positive Western blot\n6. Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1\\&2 or Syphilis serology\n7. 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\n8. Absence of adequate social support\n9. 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", "minimumAge": "18 Years", "healthyVolunteers": false }, "enrollment": { "type": "ESTIMATED", "count": 3 }, "startDate": "2020-01-10", "completionDate": "2027-06-09", "lastUpdated": "2026-02-12T00:00:00.000Z", "firstPosted": "2019-12-05T00:00:00.000Z", "resultsAvailable": false, "sourceRegistries": [ "clinicaltrials_gov" ], "sourceUrls": [ "https://clinicaltrials.gov/study/NCT04186650" ] }, "identifiers": [ { "idType": "nct", "idValue": "NCT04186650", "declaredBy": "clinicaltrials_gov" }, { "idType": "eudract", "idValue": "2016-002790-35", "declaredBy": "clinicaltrials_gov" } ], "sourceRecords": [ { "registry": "clinicaltrials_gov", "registryName": "ClinicalTrials.gov", "license": "public-domain", "sourceId": "NCT04186650", "sourceUrl": "https://clinicaltrials.gov/study/NCT04186650", "sourceStatus": "ACTIVE_NOT_RECRUITING", "retrievedAt": "2026-09-24T04:33:51.474Z", "payloadSha256": "c3a24a608bd68a56e059d3afd57bb649bf2d50e9b6876258d8fc2d8cd0c5f3e3", "piiRedacted": true } ], "citation": { "canonicalId": "NCT04186650", "sources": [ { "registry": "clinicaltrials_gov", "registryName": "ClinicalTrials.gov", "license": "public-domain", "sourceId": "NCT04186650", "verifyAt": "https://clinicaltrials.gov/study/NCT04186650", "retrievedAt": "2026-09-24T04:33:51.474Z", "payloadSha256": "c3a24a608bd68a56e059d3afd57bb649bf2d50e9b6876258d8fc2d8cd0c5f3e3", "text": "ClinicalTrials.gov, record NCT04186650 (https://clinicaltrials.gov/study/NCT04186650), retrieved 2026-09-24, licence: public-domain." } ], "crossRegistry": false, "dedupMethod": "exact cross-registry identifier matching; probabilistic = review only", "text": "Trial NCT04186650, registered in ClinicalTrials.gov, retrieved 2026-09-24. 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