Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing… (NCT05111600) | Clinical Trial Compass
TerminatedPhase 2/3
Open-label, Pivotal Clinical Trial to Confirm Efficacy and Safety of Autologous Grafts Containing Stem Cells Genetically Modified for Epidermis Restoration in Patients With Junctional Epidermolysis Bullosa
Stopped: Development program review and update due to Sponsor re-organization
France, Italy2 participantsStarted 2022-07-15
Plain-language summary
Prospective, multicenter and multinational, open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified transduced with a LAMB3-gamma retroviral vector. The purpose of this study is to demonstrate the safety and efficacy after one or more treatments with genetically corrected cultured epidermal autograft (Hologene 5) for restoration of the epidermis in patients with generalized intermediate LAMB3-dependent Junctional Epidermolysis Bullosa.
Who can participate
Age range6 Months – 65 Years
SexALL
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Inclusion criteria
✓. Signed and dated informed consent prior to any study-related procedures.
✓. Male and female patients between 6 months and 65 years old;
✓. Diagnosis of generalized intermediate LAMB3-dependent JEB, confirmed by NGS or Sanger sequencing and/or immunofluorescence;
✓. Detectable residual expression of laminin 332 (and its beta-3 chain) by immunofluorescence and/or Western Blot analysis;
✓. Presence of blisters and/or ≥ 6 cm2 erosions (persistent or recurrent for more than 3 months); the area of the erosion can be considered as the sum of smaller areas in the same body part (i.e. leg, thigh, arm, …)
✓. A cooperative attitude to follow the study procedures (caregivers in case of children);
✓. Patients' compliance with the study schedule and procedures, including complete immobilization of the transplanted areas for at least two weeks and hospitalization up to 1 month after transplantation.
Exclusion criteria
✕. Known or suspected intolerance to anaesthesia;
✕. Presence of any skin cancers in the area(s) qualified for treatment;
✕. Clinical and/or laboratory signs of acute systemic infections at the time of screening. Patient can be re-screened after appropriate treatment;
✕. Female subjects: Pregnant (as evident by a positive urine hCG or serum-hCG test) or lactating women and all women physiologically capable of becoming pregnant (i.e. women of childbearing potential \[WOCBP\]) UNLESS they are willing to use highly effective birth control methods;
✕. Allergy, sensitivity or intolerance to study medication excipients or other material required by study protocol (as per Investigator's brochure)
✕. Contraindications to the local or systemic antibiotics and/or corticosteroids foreseen by the protocol;
✕. Contraindications to undergo extensive surgical procedures;