Clinical trial NCT04692376: MSC for Treatment of cGVHD After Allo-HSCT
Official title: Mesenchymal Stem Cell for Treatment of Chronic Graft-versus-host Disease After Allogenetic Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Chronic Graft-versus-host Disease
Keywords: Hematopoietic Stem Cell Transplantation
Chronic Graft-versus-host disease
Mesenchymal Stem Cells
Lead sponsor: Nanfang Hospital, Southern Medical University
Sponsor class: OTHER
Collaborators: name: Sun Yat-sen University
name: Army Medical University, China
name: Peking University People's Hospital
First posted: 2020-12-31
Enrollment: 152
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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)

Primary outcomes:
measure: ORR
timeFrame: 12 weeks after the first dose of MSCs
Secondary outcomes:
measure: OS
timeFrame: 1 year after the first dose of MSCs
measure: DFS
timeFrame: 1 year after the first dose of MSCs
measure: Response rate
timeFrame: 4 weeks after the first dose of MSCs
measure: EBV DNA-emia
timeFrame: 1 year after the first dose of MSCs
measure: CMV DNA-emia
timeFrame: 1 year after the first dose of MSCs
measure: PGF
timeFrame: 1 year after the first dose of MSCs
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Department of Hematology,Nanfang Hospital, Southern Medical University
state: Guangdong
Study officials:
affiliation: Nanfang Hospital, Southern Medical University
name: Qi-fa Liu, MD
role: PRINCIPAL_INVESTIGATOR
References: