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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

NCT05111600 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2025-02-19

No results posted yet for this study

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.

Conditions

  • Junctional Epidermolysis Bullosa Non-Herlitz Type

Interventions

DRUG

Transplantation

Implantation of autologous cultured grafts containing genetically modified stem cells for restoration of functional skin on pre-selected areas

Sponsors & Collaborators

  • IRCCS San Raffaele

    collaborator OTHER

  • University of Modena and Reggio Emilia

    collaborator OTHER

  • Holostem s.r.l.

    lead INDUSTRY

Principal Investigators

  • Cristina Magnoni, MD, Surgeon · Struttura Complessa di Dermatologia Azienda Ospedaliero Universitaria Policlinico di Modena

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-15
Primary Completion
2024-03-12
Completion
2024-11-22

Countries

  • France
  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05111600 on ClinicalTrials.gov