Mesenchymal Stem Cells Combined With Cord Blood for Treatment of Graft Failure

Phase 2

• Conditions: Hematopoietic Stem Cell Transplantation; Mesenchymal Stem Cells; Umbilical Cord Blood; Graft Failure; Hematological Diseases
• Interventions: Biological: Mesenchymal stem cells; Biological: Mesenchymal stem cells and cord blood

• Registration Number: NCT01763099
• Lead Sponsor: Nanfang Hospital, Southern Medical University

Brief Summary

The purpose of this study is to evaluate the utility of treating patients experiencing graft failure after autologous hematopoietic stem cell transplantation with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors or mesenchymal stem cells combined with cord blood. The first objective of this study was to evaluate the effect of such treatment on graft failure, and second object was to investigate the safety of such treatment.

Detailed Description

Autologous hematopoietic stem cell transplantation (auto-HSCT) are considered the standard of care for many malignancies, such as lymphoma, myeloma and some leukemias, and so on. Graft failure after auto-HSCT is a formidable complication. It occurs in 2-9.5% of patients and is associated with considerable morbidity and mortality related to infections and hemorrhagic complications. There are various options for the management of graft failure. The most common treatment of graft failure is growth factors such as granulocyte colony-stimulating factor (G-CSF)and recombinant erythropoietin,but it usually effective in the short term and no effect on platelet counts.

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 graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation and so on.As an important source of hematopoietic stem cell, cord blood has been widely used in clinical practice. It is reported that cord blood combined with MSCs can increase engraftment after allogeneic hematopoietic stem cell transplantation. However, to our knowledge, the report about efficacy of treatment of graft that develops after auto-HSCT using expanded BM-derived MSCs from a third-party donor combined with cord blood is absent.If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent.

In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors or MSCs combined with cord blood in treating patients with graft failure after auto-HSCT.

Study Timeline

• Status Verified: 2013-01
• Study Start: 2013-01
• Study First Submitted: 2013-01-05
• Study First Submitted QC: 2013-01-05
• Study First Posted: 2013-01-08
• Last Update Submitted: 2013-01-15
• Last Update Posted: 2013-01-16
• Primary Completion: 2015-01
• Study Completion: 2016-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• A patient age of 14-65 years
• Graft failure developing after auto-HSCT
• 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)
• Patients with any conditions not suitable for the trial (investigators' decision)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Mesenchymal stem cells	Mesenchymal stem cells	Mesenchymal stem cells group refers to treatment with mesenchymal stem cells (1×10\^6 cells/kg, intravenously)
Mesenchymal stem cells and cord blood	Mesenchymal stem cells and cord blood	Mesenchymal stem cells and cord blood group refers to treatment with mesenchymal stem cells (at a dose of 1×10\^6 cells/kg) and cord blood

Primary Outcome Measures

Name	Time	Method
hematopoietic recovery	1 year	Hematopoietic reconstitution post-transplantation is defined as reconstitution of both neutrophil and platelet numbers. Neutrophil reconstitution is defined as occurring on the first 3 consecutive days with an neutrophil(NEU)\>0.5×10\^9/L, and platelet (PLT) reconstitution is defined as the first \>20×10\^9/L for 3 consecutive days.

Secondary Outcome Measures

Name	Time	Method
infections, primary underlying disease relapse and any toxic side effects of MSCs treatment	1 year	Infections will be mainly focused within the first 100 days after MSCs treatment. Toxic side effects of treatment includes acute toxicity and late side effects. Acute toxicity principally involves the heart,live and kidney. Late toxic side effects involves principally the development of secondary tumors and relapse of the primary disease.

Trial Locations

Locations (1)

Department of Hematology,Nanfang Hospital, Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China