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

Myelodysplastic Syndromes Clinical Trial

Official title:

Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat RA and RARS of MDS

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01129739
• Other study ID #: CZheng
• Secondary ID: No. 30670903
• Status: Recruiting
• Phase: Phase 2
• First received: May 17, 2010
• Last updated: May 24, 2010
• Start date: May 2010
• Est. completion date: May 2013

Study information

• Verified date: May 2010
• Source: Shandong University
• Contact: chengyun zheng, PhD
• Phone: +86-531-85875635
• Email: chengyun.zheng[@]ki.se
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: May 2013
• Est. primary completion date: May 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Preleukemia

Intervention

Other:
• Human umbilical cord-derived MSCs
1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle
• cyclosporine A (CsA)
CsA 5mg/kg po for 6 months

Locations

Country	Name	City	State
China	Department of Hematology of the 2nd Hospital of Shandong University	Jinan	Shandong

Sponsors (2)

Lead Sponsor	Collaborator
Shandong University	National Natural Science Foundation of China

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	MDS clinical symptoms (mainly anemia symptoms)	Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.	1 year	Yes
Primary	A routine blood test	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.	1 year	Yes
Primary	Bone borrow cytomorphologic examination	Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.	1 year	Yes
Secondary	Percentage of T regulatory cell population in peripheral blood	Percentage of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.	1 year	Yes