A prospective randomized controlled study of multi-intravenous infusion of umbilical cord mesenchymal stem cells in patients with heart failure and reduced ejection fraction (PRIME-HFrEF) trial: Rationale and design

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Contemporary Clinical Trials Communications Pub Date : 2024-08-12 DOI:10.1016/j.conctc.2024.101350

Xin Gong , Yuheng Jiao , Hao Hu , Rongzhen Zhang , Wenwen Jia , Jun Zhao , Zhongmin Liu , Yuanfeng Xin , Wei Han

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Abstract

Background and objective

The use of mesenchymal stem cells for heart failure treatment has gained increasing interest. However, most studies have relied on a single injection approach, with no research yet confirming the effects of multiple administrations. The present trial aims to investigate the safety and efficacy of multi-intravenous infusion of umbilical cord-mesenchymal stem cells (UC-MSCs) in patients with heart failure and reduced ejection fraction (HFrEF).

Methods

The PRIME-HFrEF trial is a single-center, prospective, randomized, triple-blinded, placebo-controlled trial of multi-intravenous infusion of UC-MSCs in HFrEF patients. A total of 40 patients meeting the inclusion criteria for HFrEF were enrolled and randomized 1:1 to the MSC group or the placebo group. Patients enrolled will receive intravenous injections of either UC-MSCs or placebo every 6 weeks for three times. Both groups will be followed up for 12 months. The primary safety endpoint is the incidence of serious adverse events. The primary efficacy endpoint is a change in left ventricular ejection fraction (LVEF) measured by left ventricular opacification (LVO) with contrast echocardiography and magnetic resonance imaging (MRI) at 12 months. The secondary endpoints include a composite of the incidence of death and re-hospitalization caused by heart failure at the 12th month, serum NT-proBNP, growth stimulation expressed gene 2 (ST₂), and a change of right ventricular structure and function.

Conclusions

The PRIME-HFrEF study is designed to shed new light on multiple UC-MSC administration regimens for heart failure treatment.

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心力衰竭和射血分数降低患者多静脉输注脐带间充质干细胞的前瞻性随机对照研究（PRIME-HFrEF）试验：原理与设计

背景和目的间充质干细胞用于治疗心力衰竭的兴趣日益浓厚。然而，大多数研究都依赖于单次注射方法，尚未有研究证实多次给药的效果。方法PRIME-HFrEF试验是一项单中心、前瞻性、随机、三重盲、安慰剂对照试验，在HFrEF患者中多次静脉注射脐带间充质干细胞（UC-MSCs）。共有 40 名符合 HFrEF 纳入标准的患者入选，并按 1:1 随机分配到间充质干细胞组或安慰剂组。入组患者将接受静脉注射 UC 间充质干细胞或安慰剂，每 6 周一次，共注射 3 次。两组患者都将接受 12 个月的随访。主要安全性终点是严重不良事件的发生率。主要疗效终点是12个月时通过对比超声心动图和磁共振成像（MRI）测量的左心室射血分数（LVEF）的变化。次要终点包括第 12 个月时因心衰死亡和再次住院的发生率、血清 NT-proBNP、生长刺激表达基因 2 (ST2) 以及右心室结构和功能的变化。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Contemporary Clinical Trials Communications Pharmacology, Toxicology and Pharmaceutics-Pharmacology

CiteScore

2.70

自引率

6.70%

发文量

146

审稿时长

20 weeks

期刊介绍： Contemporary Clinical Trials Communications is an international peer reviewed open access journal that publishes articles pertaining to all aspects of clinical trials, including, but not limited to, design, conduct, analysis, regulation and ethics. Manuscripts submitted should appeal to a readership drawn from a wide range of disciplines including medicine, life science, pharmaceutical science, biostatistics, epidemiology, computer science, management science, behavioral science, and bioethics. Contemporary Clinical Trials Communications is unique in that it is outside the confines of disease specifications, and it strives to increase the transparency of medical research and reduce publication bias by publishing scientifically valid original research findings irrespective of their perceived importance, significance or impact. Both randomized and non-randomized trials are within the scope of the Journal. Some common topics include trial design rationale and methods, operational methodologies and challenges, and positive and negative trial results. In addition to original research, the Journal also welcomes other types of communications including, but are not limited to, methodology reviews, perspectives and discussions. Through timely dissemination of advances in clinical trials, the goal of Contemporary Clinical Trials Communications is to serve as a platform to enhance the communication and collaboration within the global clinical trials community that ultimately advances this field of research for the benefit of patients.