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Gene Therapy Trial Report

Summary

Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts


NCTID NCT04186650 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term DOID:4959
Compound Name EF1a-COL7A1-SIN retroviral vector transduced autologous skin
Sponsor Institut National de la Santé Et de la Recherche Médicale, France
Funder Type Other gov
Recruitment Status
Active not recruiting
Enrollment Count 3 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant COL7A1
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Skin graft
Drug Product Type Autologous cells
Target Tissue/Cell Keratinocytes
Delivery System Viral transduction
Vector Type RV
Editor Type none
Dose 1 Up to 6 grafts of 50 cm^2 each

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2019-11-25
Completion Date 2027-06-09
Last Update 2026-02-12

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations 2. Reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) 3. A reduced number of/or morphologically abnormal anchoring fibrils confirmed by TEM 4. Detection of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot (WB) analysis 5. Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting 6. Ability to undergo anaesthesia for skin grafting procedures 7. Subjects aged 18 years, willing and able to give informed consent Exclusion Criteria: 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links