MSCs With or Without Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment

Hematological Diseases Clinical Trial

Official title:

Mesenchymal Stem Cells With or Without G-CSF Mobilized Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplant

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02240992
• Other study ID #: NFH-PBSC-MSC-PGF-2014
• Status: Recruiting
• Phase: Phase 2/Phase 3
• First received: September 13, 2014
• Last updated: September 13, 2014
• Start date: September 2014
• Est. completion date: December 2018

Study information

• Verified date: September 2014
• Source: Nanfang Hospital of Southern Medical University
• Contact: Ren Lin, MD
• Phone: +86-020-62787883
• Email: lansinglinren[@]hotmail.com
• Is FDA regulated: No
• Health authority: China: Ethics Committee
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to compare the efficacy of mesenchymal stem cells (MSCs) with or without granulocyte colony-stimulating factor (G-CSF) mobilized peripheral stem cells (PBSC) in treating patients experiencing poor graft function or delayed platelet engraftment after allogeneic hematopoietic stem cell transplantation.

Description:

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) is the only cure for many hematologic diseases. However, about 5-27% of patients would suffer from poor graft function (PGF) and more recipients might develop delayed platelet engraftment (DPE) after allo-HSCT. These complications are associated with considerable mortality related to infections or hemorrhagic complications. Treatment of PGF and DPE usually involves hematopoietic growth factors such as granulocyte colony-stimulating factor (G-CSF) and thrombopoietin (TPO), or second transplantation, but these methods have dismal effect or even a significant risk of graft-versus-host disease (GVHD).

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 include improving hematopoietic engraftment, preventing and treating graft-versus-host disease after allo-HSCT and so on. Some studies have shown that MSCs combined with PBSC or cord blood could be useful to improve engraftment after HSCT. Several reports suggested MSCs might be effective in the treatment of PGF.

However, the efficacy of MSCs as single-drug treatment for PGF or DPE is unsatisfactory in our previous study. Therefore, in the present study, G-CSF mobilized PBSC will be used combined with MSCs in the patients with PGF or DPE after allo-HSCT.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: December 2018
• Est. primary completion date: September 2018
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 14 Years to 65 Years

Eligibility

Inclusion Criteria:

• Age:14-65 years

• PGF or DPE after allo-HSCT

• Subjects (or their legally acceptable representatives) must have signed an informed consent document

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

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Delayed Platelet Engraftment
• Hematologic Diseases
• Hematological Diseases
• Poor Graft Function
• Stem Cell Transplantation, Hematopoietic

Intervention

Biological:
• PBSC

• MSCs

Locations

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

Sponsors (11)

Lead Sponsor

Collaborator

Nanfang Hospital of Southern Medical University

Academy Military Medical Science, China, Guangdong General Hospital, Guangzhou First Municipal People’s Hospital, Guangzhou General Hospital of Guangzhou Military Command, Huazhong University of Science and Technology, Peking University People's Hospital, Southern Medical University, China, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Sun Yat-sen University, Third Affiliated Hospital, Sun Yat-Sen University

Country where clinical trial is conducted

China, 

References & Publications (2)

Meuleman N, Tondreau T, Ahmad I, Kwan J, Crokaert F, Delforge A, Dorval C, Martiat P, Lewalle P, Lagneaux L, Bron D. Infusion of mesenchymal stromal cells can aid hematopoietic recovery following allogeneic hematopoietic stem cell myeloablative transplant: a pilot study. Stem Cells Dev. 2009 Nov;18(9):1247-52. doi: 10.1089/scd.2009.0029. — View Citation

Sánchez-Guijo FM, López-Villar O, López-Anglada L, Villarón EM, Muntión S, Díez-Campelo M, Perez-Simón JA, San Miguel JF, Caballero D, del Cañizo MC. Allogeneic mesenchymal stem cell therapy for refractory cytopenias after hematopoietic stem cell transplantation. Transfusion. 2012 May;52(5):1086-91. doi: 10.1111/j.1537-2995.2011.03400.x. Epub 2011 Oct 24. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants with Hematopoietic Recovery	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.	1 year	No
Secondary	Incidence of graft-versus-host disease	Graft-versus-host disease (GVHD) includes acute GVHD and chronic GVHD.	1 year	No
Secondary	Incidence of infections	Infections include bacterial, invasive fungal and viral infections	1 year	No
Secondary	Incidence of primary underlying disease relapse		1 year	No
Secondary	Incidence of acute toxicity	Acute toxicity mainly involves the heart,live and kidney.	up to 100 days	Yes