EUCTR2016-002790-35-FR
Active, not recruiting
Phase 1
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
ConditionsThe trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL7A1 cDNA.MedDRA version: 20.0 Level: LLT Classification code 10074980 Term: Epidermolysis bullosa aquisita System Organ Class: 100000004858Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- The trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL7A1 cDNA.
- Sponsor
- INSERM
- Enrollment
- 3
- Status
- Active, not recruiting
- Last Updated
- 6 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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:
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
Outcomes
Primary Outcomes
Not specified
Similar Trials
Active, not recruiting
Phase 1
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseSickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]EUCTR2019-000331-63-FRbluebird bio, Inc.35
Active, not recruiting
Phase 1
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseEUCTR2019-000331-63-NLbluebird bio, Inc.35
Active, not recruiting
Phase 1
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseSickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]EUCTR2019-000331-63-ITBLUEBIRD BIO, INC.35
Active, not recruiting
Phase 1
Clinical study to assess the efficacy and safety of gene therapy for the treatment of Sickle Cell DiseaseSickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]EUCTR2019-000331-63-GBbluebird bio, Inc.35
Completed
Phase 1
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent PorphyriaAcute Intermittent PorphyriaNCT02082860Digna Biotech S.L.8