Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Myelodysplastic Syndromes

Phase 2

• Conditions: Myelodysplastic Syndromes
• Interventions: Other: Human umbilical cord-derived MSCs; Other: cyclosporine A (CsA)

• Registration Number: NCT01129739
• Lead Sponsor: Shandong University

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg on the subjects for refractory anemia (RA) and refractory anemia with ring sideroblast (RARS) of myelodysplastic syndromes (MDS).

Detailed Description

Myelodysplastic syndromes are bone marrow stem cell disorders resulting in disorderly and ineffective hematopoiesis. MDS is characterized by variable degrees of cytopenias (anemia, neutropenia, and thrombocytopenia ) and risk of transformation to leukemia.

To date treatment of MDS is unsatisfactory: chemotherapy has a limited role in the management of leukemic progression; autologous stem cell transplantation does not prolong relapse-free survival and stem cell transplantation is poorly tolerated in older individuals. Some MDS patients have been shown to respond to a wide variety of immunosuppressive agents ranging from corticosteroids to CsA and antithymocyte globulin (ATG). However, the overall response rate is less than 30%. In fact, few treatments appear to change the natural history of MDS.

The management of MDS patients therefore remains to be improved. Human MSCs isolated from Wharton's jelly of the umbilical cord/placenta have been shown to have immunosuppressive, stimulating hematopoiesis and tissue repairing properties. This study will evaluate the safety and effectiveness of MSC transplant in the MDS patients.

This study will last about 3 years. Participants will be randomly assigned to receive either MSC transplant (Group 1) or CsA therapy alone (Group 2). Patients will undergo MSC transplant at the start of the study (defined as Day 0). After 3 months, patients will receive the second MSC transplantation when one responds well to the treatment. After 3, 6 and 12 months from the first transplantation, patients will be evaluated.

Study Timeline

• Status Verified: 2010-05
• Study Start: 2010-05
• Study First Submitted: 2010-05-17
• Study First Submitted QC: 2010-05-24
• Last Update Submitted: 2010-05-24
• Study First Posted: 2010-05-25
• Last Update Posted: 2010-05-25
• Primary Completion: 2013-05
• Study Completion: 2013-05

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Patient age 18~80 years old with plan to infuse MSCs.
2. Histologically documented or cytologically confirmed diagnosis of MDS with WHO classification of MDS-RA and MDS-RARS.
3. Patients must have an ECOG 0~2.
4. No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 mmol/L.
5. No active severe viral or fungus infection.
6. Each patient must sign written informed consent.

Exclusion Criteria

1. Psychiatric condition that would limit informed consent.
2. HIV positive
3. Positive Pregnancy Test
4. Patient has enrolled another clinical trial study within last 4 weeks.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Human umbilical cord-derived MSCs	Human umbilical cord-derived MSCs	Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle
cyclosporine A (CsA)	cyclosporine A (CsA)	CsA at a dose of 5 mg CsA/kg

Primary Outcome Measures

Name	Time	Method
MDS clinical symptoms (mainly anemia symptoms)	1 year	Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.
Bone borrow cytomorphologic examination	1 year	Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.
A routine blood test	1 year	A routine blood test, which contains WBC, Neu, RBC, Hb and PLT, will be mainly tested in every month after transplanting MSCs for one year.

Secondary Outcome Measures

Name	Time	Method
Percentage of T regulatory cell population in peripheral blood	1 year	Percentage of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.

Trial Locations

Locations (1)

Department of Hematology of the 2nd Hospital of Shandong University; 🇨🇳 Jinan, Shandong, China