NCT04153630

Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Instituto de Investigación Hospital Universitario La Paz · Academic / Other
• Sex: All
• Age: 12 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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

Conditions

• Epidermolysis Bullosa Dystrophica, Recessive

Interventions

Type	Name	Description
BIOLOGICAL	mesenchymal stem cells derived from bone marrow (BM-MSCs)	Procedure: Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg

Timeline

• Start date: 2018-05-17
• Primary completion: 2021-03-01
• Completion: 2021-03-01
• First posted: 2019-11-06
• Last updated: 2019-11-08

Locations

1 site across 1 country: Spain