Adipose-derived Stem Cells Therapy for Moderate Chronic Obstructive Pulmonary Disease in Adult Subjects: A Phase III Randomized, Multicenter Trial: RESPIRE Protocol
Authors:   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Monterroso Yancor Bryan ,   Universidad Francisco Marroquín, Facultad Medicina, Ciudad de Guatemala, Guatemala, Basegoda Curiel Wilhelm Sebástian ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States,   Universidad Francisco Marroquín, Facultad Medicina, Ciudad de Guatemala, Guatemala, Lemus-Martin Roselyn ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Sanchez Iriarte Nicole Alejandra ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Ribeiro Maíra ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Bothou Christina ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Alonso-Ruiz Daniela ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Reger Mira ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Rosales-Salán Mónica Elizabeth ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Maffioletti Gonçalves Lucas ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Kim Hee-Young ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Bustos José ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Wong-Valenzuela Emilio Israel ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, Chavez Fernandez Diego Rolando ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States, De Maria Maddalena ,   Principles and Practice in Clinical Research, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, United States
Published: April 2024

Introduction: Chronic obstructive pulmonary disease (COPD) is one of the leading causes of death worldwide and has a prevalence of 11.7%. Current treatment focuses on reducing symptoms and exacerbations, as no definitive intervention exists. Regenerative treatments such as mesenchymal stem cell (MSCs) therapy have shown promise in preclinical and clinical studies. This article presents a study protocol to evaluate the efficacy of adipose-derived (AD) MSCs therapy in improving FEV-1 in COPD patients. Methods: A phase III, multicenter, randomized, double-blind, placebo-controlled study is proposed to test the clinical safety and efficacy of AD-MSC therapy in patients with COPD. The population is subjects with COPD Global Initiative for Chronic Obstructive Lung Disease (GOLD) 2 (50%≤FEV1<80% predicted) and Group D (two exacerbations or one hospitalization and mMRC>2 or CAT>10). This study aims to evaluate the improvement of FEV1 from baseline to 90 days after AD-MSC infusion. The primary outcome is FEV1-improvement (%) over the baseline for the treatment and placebo groups, assessed on study day 90 vs. baseline (day 0). For the primary outcome, the statistical analysis will use the Mann-Whitney U test to analyze the FEV1 for the treatment and control groups at 90 days against the baseline. Discussion: Aside from symptomatic relief, there is a growing demand for a curative treatment for COPD. The potential of novel AD-MSCs therapies for COPD improvement is currently in early development. Preclinical, phase I, and a few phase II studies for AD-MSCs as a COPD treatment have proven optimistic results. This phase III, randomized, multicenter trial proposes evaluating an alternative treatment for this condition that may improve lung function.