Recessive Dystrophic Epidermolysis Bullosa Clinical Trial
— HOLOGENE7Official title:
Prospective, Open-label, Uncontrolled Clinical Trial to Assess the Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified With a Gamma-retroviral (rv) Vector Carrying COL7A1 cDNA for Restoration of Epidermis in Patients With Recessive Dystrophic Epidermolysis Bullosa.
Verified date | February 2022 |
Source | Holostem Terapie Avanzate s.r.l. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying COL7A1 complementary DNA (cDNA) for restoration of the epidermis in patients with recessive dystrophic epidermolysis bullosa. The purpose of this study is to demonstrate the safety and efficacy after one or more treatments with genetically corrected cultured epidermal autograft (Hologene 7) in patients suffering of recessive dystrophic epidermolysis bullosa (RDEB) with COL7A1 mutation.
Status | Terminated |
Enrollment | 3 |
Est. completion date | August 6, 2018 |
Est. primary completion date | August 6, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years to 54 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Austria | EB House Austria, Department of Dermatology, Paracelsus Medical University | Salzburg |
Lead Sponsor | Collaborator |
---|---|
Holostem Terapie Avanzate s.r.l. | Paracelsus Medical University |
Austria,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Treatment success | Percentage of patients defined as "success" by Investigator site according to the same parameters as for the primary and key secondary efficacy assessments 12 months after last treatment; | 12-months | |
Other | Fibrin re-absorption | • Complete matrix re-absorption (by visual inspection) one week after the transplantation and clinical success (defined as the primary efficacy assessment) at early assessment time points (1 and 4 weeks after transplantation). | 1- and 4-weeks | |
Primary | Safety | number and percentage of patients experiencing treatment-related adverse events (TRAEs), serious adverse events (SAEs) and serious adverse drug reactions (ADRs) up to 3 months after the first treatment. | 3-month | |
Secondary | Efficacy | Percentage of patients with clinical success after one or more treatments with study product at 3 and 12 months follow up.
Clinically success is reached when both the following conditions are met: o Regeneration of a clinically normal appearing skin with absence of detectable blister. AND o Restoration of type VII collagen expression and restoration of anchoring fibrils in the treated area. |
3- and 12-months |
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