Overview

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

Status:
Not yet recruiting

Trial end date:
2023-06-01

Target enrollment:

Participant gender:

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.

Phase:

Phase 2/Phase 3

Details

Lead Sponsor:

Holostem Terapie Avanzate s.r.l.

Collaborators:

IRCCS San Raffaele
University of Modena and Reggio Emilia