MSC for Treatment of cGVHD After Allo-HSCT

Chronic Graft-versus-host Disease Clinical Trial

Official title:

Mesenchymal Stem Cell for Treatment of Chronic Graft-versus-host Disease After Allogenetic Hematopoietic Stem Cell Transplantation

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04692376
• Other study ID #: MSC-cGVHD-2020
• Status: Recruiting
• Phase: Phase 2
• Start date: January 10, 2021
• Est. completion date: June 30, 2023

Study information

• Verified date: December 2020
• Source: Nanfang Hospital of Southern Medical University
• Contact: Ren Lin, MD
• Phone: +86-020-62787883
• Email: lansinglinren[@]hotmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy of mesenchymal stem cells in patients with chronic graft-versus-host disease.

Description:

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) can cure many hematologic diseases. Although great progress has been made in the prevention and treatment of side effects associated with transplantation,chronic graft-versus-host disease(cGVHD) remains an important complication that occurs in about 50% patients. The mortality of cGVHD and its complication could reach up to 50%,and cGVHD seriously influence the quality of life. At present, the first line treatment of cGVHD remains in discussion. Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating GVHD after allo-HSCT and so on. However, the efficacy of treatment of cGVHD remains undetermined. In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded MSCs in treating patients with cGVHD.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 152
• Est. completion date: June 30, 2023
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

• A patient age of 18-65 years
• Recipients of allogeneic hematopoietic stem cell transplantation Patients with moderate/ severe cGVHD without systemic treatment
• Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

Exclusion Criteria:

• Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
• Primary disease relapse
• Expected lifetime less than 3 months
• Patients with any conditions not suitable for the trial (investigators' decision)

Related Conditions & MeSH terms

• Chronic Graft-versus-host Disease
• Graft vs Host Disease

Intervention

Biological:
• Mesenchymal stem cells
Mesenchymal stem cells (MSCs) will be intravenously infused via a central venous catheter(at a dose of 1×10^6 cells/kg, over 15 mins) weekly. MSCs will be administrated for 8 doses.

Drug:
• Glucocorticoids
Glucocorticoids (i.e. Methylprednisolone) will be used with an initial dose of 1mg/kg.
• cyclosporine
Cyclosporine (CsA) will be used with an initial dose of 2.5mg/kg/d and adjusted according to the concentration of CsA. The targeted concentration is 200-300 ng/Ml.

Locations

Country	Name	City	State
China	Department of Hematology,Nanfang Hospital, Southern Medical University	Guangzhou	Guangdong

Sponsors (4)

Lead Sponsor	Collaborator
Nanfang Hospital of Southern Medical University	Peking University People's Hospital, Sun Yat-sen University, Xinqiao Hospital, Army Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ORR	Overall response rate (ORR)includes complete response (CR) and part response (PR).	12 weeks after the first dose of MSCs
Secondary	OS	Overall survival (OS)	1 year after the first dose of MSCs
Secondary	DFS	Disease-free survival (DFS)	1 year after the first dose of MSCs
Secondary	Response rate		4 weeks after the first dose of MSCs
Secondary	EBV DNA-emia	EBV DNA-emia refers to detection of EBV DNA in peripheral blood via PCR.	1 year after the first dose of MSCs
Secondary	CMV DNA-emia	CMV DNA-emia refers to detection of CMV DNA in peripheral blood via PCR.	1 year after the first dose of MSCs
Secondary	PGF	Poor graft function (PGF) refers to a slow or incomplete recovery of blood cell counts (ANC =0.5x10^9/L and PLT =20x10^9/L) by +28 days after allo-HSCT or a fall in blood cell counts to levels fulfilling the diagnostic criteria for PGF after successful and prompt hematopoietic engraftment.	1 year after the first dose of MSCs