Design and rationale of CATO, a Phase IIA, randomized, double-blind, placebo-controlled study of single or repeated intravenous administration of umbilical cord-derived mesenchymal stromal cells in ischemic cardiomyopathy

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RATIONALE: To date, almost all studies of cell therapy in chronic heart failure (HF) have delivered cells transendocardially or intracoronarily and all have used a single cell dose, which limits therapeutic efficacy because transplanted cells disappear rapidly. Repeated administrations are necessary to replace the cells that disappear, but this is difficult or impossible when cells are delivered invasively. Further, transendocardial delivery is not feasible in many patients, carries risks, and requires specialized training and equipment, thereby limiting its widespread applicability. Intravenous infusion of cells is cheaper, simpler, safer, and less invasive; most importantly, it enables administration of multiple cell doses. PRIMARY GOAL: The CATO trial tests a new strategy-repeated intravenous infusions of cells in chronic HF. The goal is to determine whether intravenous cell therapy is beneficial and whether multiple cell doses are superior to a single dose. This trial is the culmination of a translational journey that began more than a decade ago in animal models of HF. DESIGN: CATO is a Phase IIA randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy of intravenous infusion of umbilical cord-derived mesenchymal stromal cells (UC-MSCs) in patients with ischemic HF. Sixty subjects will be randomized to 3 groups: control (placebo), 1 dose of UC-MSCs, or 4 repeated doses of UC-MSCs, with a comprehensive evaluation of cardiac function and structure, functional capacity, quality of life, and biomarkers over 12 months. ENROLLMENT DATES AND CURRENT STATUS: CATO began enrollment on March 4, 2024. As of November 30, 2025, a total of 47 patients have been enrolled. Enrollment is expected to be completed by March 2026, and follow-up by March 2027. SIGNIFICANCE: CATO is the first trial of UC-MSCs for HF in the US, the first study of intravenous cell therapy for ischemic HF in the US, and the first randomized, double-blind, placebo-controlled trial of repeated cell doses for chronic HF. The 2 strategies tested in CATO (intravenous cell delivery and repeated doses) have the potential to be important advances in the management of HF CLINICAL TRIAL REGISTRATION: Clinicaltrials.gov, NCT06145035 https://clinicaltrials.gov/study/ NCT06145035.

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