Abstract
Abstract
Background
Systemic corticosteroid therapy failure is quite common in patients with newly diagnosed acute graft-versus-host disease (aGVHD). Growing evidence has suggested that mesenchymal stem cell (MSC) therapy could be a promising treatment option for aGVHD due to its distinctive immunomodulating functions. However, there is a lack of randomized well-controlled clinical trials.
Methods
This is a clinical trial protocol for a multicenter, randomized, double-blind, placebo-controlled phase II study. The aim of the trial is to evaluate the efficacy and safety of the administration of the human umbilical cord-derived MSC product hUC-MSC PLEB001 in patients with grade II–IV, steroid-refractory aGVHD. A total of 96 patients will be randomized 1:1 to receive MSC or placebo treatment twice per week for 4 weeks, in addition to second-line therapy according to institutional standards. Patients who achieve partial response (PR) at day 28 will be eligible to receive further infusions twice per week for an additional 4 weeks.
Discussion
This study will evaluate the efficacy and safety of MSC therapy in patients who have failed first-line steroid treatment for grade II–IV aGVHD.
Trial registration
Chinese Clinical Trial Registry (ChiCTR), ChiCTR2000035740. Registered on 16 August 2020.
Funder
National Natural Science Foundation of China
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Medicine (miscellaneous)
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