Epidermolysis Bullosa Dystrophica, Recessive Clinical Trial
Official title:
Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process and / or the mucocutaneous fragility phenotype associated with EBDR.
The Main Objective is to evaluate the safety and therapeutic efficacy of haploidentical MSCs
derived from bone marrow administered by intravenous injection for the treatment of patients
with RDBS. The assessment of the symptomatic improvement of the treated patients will be made
regarding the baseline situation and the response to treatment at the biochemical,
histological and molecular level.
Secondary Objectives:
Describe the clinical and molecular phenotype of the mucocutaneous involvement of patients,
including the characterization of the mutations responsible for the disease.
Study drug: Allogenic mesenchymal cells (haploidentical) derived from bone marrow and
expanded.
Method of administration: Systemic / Intravenous Administration dose: 2-3x10e6 BM-MSC / Kg.
Weekly dose for three consecutive weeks
Follow-up period: 12 months after the infusion. However, patients will be monitored outside
the clinical trial over a 5-year period
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