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

Phase 1/2 Unknown 9 enrolled Instituto de Investigación Hospital Universitario La Paz CIBER Enfermedades raras · collab DEBRA · collab Instituto de Salud Carlos III · collab St John's Institute of Dermatology Kings College London · collab Universidad Carlos III Madrid (TERMeG) · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2018-05-17
Primary completion: 2021-03
Completion: 2021-03

Publications

Maseda R, Arriba MC, Martinez-Santamaria L, Jimenez E, Herraiz-Gil S, Illera N, Quintana-Castanedo L, Garcia M, Suarez-Sancho S, Yanez R, Perez-Conde I, Carretero M, Martinez-Queipo M, de Paz R, Leon C, Jimenez-Yuste V, Borobia AM, Vicente A, Lwin SM, McGrath JA, Fernandez-Santos ME, Butta N, Sacedon R, Del Rio M, de Lucas R, Escamez MJ. MesenSistem-EB: systemic haploidentical mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa associated with clinical benefits and correlated with MCP1 and sCD40L dynamics. Front Immunol. 2026 May 5;17:1789537. doi: 10.3389/fimmu.2026.1789537. eCollection 2026.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	mesenchymal stem cells derived from bone marrow	Cell therapy	2e+06 cells/kg	Intravenous
Subject	mesenchymal stem cells derived from bone marrow	Cell therapy	3e+06 cells/kg	Intravenous

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

Summary

Timeline

Publications

Drugs

Indications