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Gene Therapy Trial Report

Summary

Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.


NCTID NCT02984085 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term DOID:4959
Compound Name HOLOGENE7
Compound Description Genetically corrected cultured epidermal autograft (ATMP)
Sponsor Holostem s.r.l.
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 3 (ACTUAL)
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 Epidermal cells
Delivery System Viral transduction
Vector Type RV
Dose 1 Transduced autologous skin grafts

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2016-12-02
Completion Date 2018-08-06
Last Update 2022-02-16

Participation Criteria


Eligible Age 6 Years - 54 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Signed and dated informed consent prior to any study-related procedures. Informed consent will also include the possibility of additional transplantations and of the rolling over to the long-term extension period; 2. Adult male and female patients (≥18 years old and \< 55); Paediatric patients aged 6 to 17 years will be also enrolled. 3. RDEB molecular characterization by mutation analysis; 4. Non-collagenous domain (NC1 or NC2) antibody immunofluorescence or staining positive in Western Blot; 5. Presence of chronic (persistent for more than 3 months) large wounds (\>10 cm2) and/or erosion; 6. A cooperative attitude to follow up the study procedures (Caregivers in case of minors). Exclusion Criteria: 1. Known or suspected intolerances against anaesthesia; 2. Bad general condition (ECOG index \>1) 3. Unresectable or metastasizing squamous cell carcinoma (SCCs); 4. Antibodies to type VII collagen associated antigens demonstrated on indirect immunofluorescence; 5. Clinical and/or laboratory signs of acute systemic infections at the time of screening. Patient can be re-screened after appropriate treatment; 6. Severe systemic diseases (i.e. uncompensated diabetes); 7. Female subjects: pregnant or lactating women and all women physiologically capable of becoming pregnant (i.e. women of childbearing potential) UNLESS they are willing to use one or more reliable methods of contraception with a Pearl index ≤1. 8. Allergy, sensitivity or intolerance to drugs or excipients (hypersensitivity to any of the excipients listed in Investigator's brochure or in this protocol): * Transport medium (Dulbecco's Modified Eagles Medium supplemented with L-glutamine) * Fibrin support * Betaisodona 9. Contraindications to the local or systemic antibiotics and/ or corticosteroids foreseen by the protocol; 10. Contraindications to undergo extensive surgical procedures; 11. Clinically significant or unstable concurrent disease or other clinical contraindications to stem cell transplantation based upon investigator's judgment or other concomitant medical conditions affecting grafting procedure; 12. Patients (or parents in case of paediatric subject) unlikely to comply with the study protocol or unable to understand the nature and scope of the study or the possible benefits or unwanted effects of the study procedures and treatments. 13. Participation in another clinical trial where investigational drug was received less than 6 months prior to screening visit.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations Austria

Regulatory Information


Has US IND False
FDA Designations
Recent Updates 3 patients enrolled, study terminated, to be replaced by a new study

Resources/Links


Resources/Links

No External Links Available.