NCT05531266

Umbilical Cord Mesenchymal Stem Cells as First-line Treatment for Patients With Acute Graft Versus Host Disease

Status unknown NA Last updated 7 September 2022

What this trial tests

NA trial testing hUC-MSCs in aGVHD in 182 participants. Status unknown.

Timeline

1 September 2022

Primary endpoint
1 March 2024

1 March 2025

Quick facts

Lead sponsor	Institute of Hematology & Blood Diseases Hospital, China
Phase	NA
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	182
Start date	1 September 2022
Primary completion	1 March 2024
Estimated completion	1 March 2025
Sites	1 location across China

Drugs / interventions tested

• hUC-MSCs — full drug profile →
• glucocorticoids — full drug profile →

Conditions studied

• aGVHD — all drugs for aGVHD →

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Who can join

Eligibility, any sex, with aGVHD. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Allogenic haemopoietic stem cell transplantation (allo-HSCT) is the effective treatment for many hematologic malignancies and some non-malignant diseases. In recent years, with the rapid improvement of economy and medical level, the number of cases of hematopoietic stem cell transplantation (HSCT) develops rapidly in China. In 2019, 12,323 cases of HSCT were completed in China, with allo-HSCT accounting for 9600 cases of which.

However, Graft versus host disease (GVHD) is one of the most common and serious complications after Allo-HSCT. The incidence of acute GVHD (aGVHD) is as high as 40%-60% in HLA-matched sibling transplantation, and the incidence is even higher in haplo-hematopoietic stem cell transplantation(haplo-HSCT) and unrelated donor transplantation. By Glucksberg grading standard, the 5-year survival rates of grade III and IV aGVHD are 25% and 5% respectively, indicating severe GVHD directly affects the survival of Allo-HSCT patients.

The first-line treatment for aGVHD is still glucocorticoid, while the effective rate is only 30%-50%. Moreover, due to immunosuppression and increasing risk of infection, the efficacy of second-line treatments including polyclonal antibodies, monoclonal antibodies, immunosuppressants, immunotoxins, chemotherapy drugs, and light therapy for steroid resistant aGVHD is also poor, with the overall survival rate of 5%-30%.

Mesenchymal stem cells (MSCs) are multipotent cells, which can promote engraftment and hematopoietic reconstruction by secreting a variety of hematopoietic promoting factors, expressing adhesion molecules supporting hematopoietic stem cells, guiding homing of hematopoietic stem cells and providing hematopoietic microenvironment. At the same time, MSCs can modulate immune responses by affecting the proliferation of T cells and the migration of T cells and DC, inducing the expansion of Treg cells, inhibiting the secretion of antibodies by B lymphocytes, and regulating the secretion of soluble factors such as NO and IDO. As a result of these characteristics and the poor immunogenicity, MSCs are a promising alternative treatment for GVHD.

Currently, UK and EU guidelines has recommended MSC as a third-line treatment for grade 2-4 acute GVHD, and the safety and efficacy of umbilical cord derived MSCs in the prevention and treatment of GVHD has also been reported by several transplantation centers in China.However, MSCs have not been used for first-line treatment of aGVHD. Therefore, the investigators designed this study to evaluate the safety and efficacy of UC-derived MSCs as the first line treatment in patients with aGVHD.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Extracellular vesicles: a new frontier in diagnosing and treating graft-versus-host disease after allogeneic hematopoietic cell transplantation.
   Wu P, Wang Z, Sun Y, Cheng Z, et al · · 2025 · cited 5× · PMID 40133949 · DOI 10.1186/s12951-025-03297-y
2. Modular mastery of inflammation: umbilical cord mesenchymal stem cells as a therapeutic frontier.
   Yin L, Sun CY, Chen GL, Xiang Z, et al · · 2025 · PMID 41488628 · DOI 10.3389/fimmu.2025.1721947
3. The roles and clinical applications of mesenchymal stem cells and their exosomes in hematologic diseases.
   Deng M, Zhang X, Zhang Y. · · 2025 · PMID 41444972 · DOI 10.1186/s13287-025-04880-8
4. Mesenchymal Stromal Cells: Bridging the Gaps in Hematologic Disease Therapy.
   Zhao X, Ming X, Wu J, Zhu X, et al · · 2026 · PMID 41254448 · DOI 10.1007/s12015-025-11011-6