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

Phase 2
18 Years
80 Years
Open (Enrolling)
Myelodysplastic Syndromes

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Trial Information

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

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.

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

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.

Type of Study:


Study Design:

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

Outcome Measure:

MDS clinical symptoms (mainly anemia symptoms)

Outcome Description:

Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.

Outcome Time Frame:

1 year

Safety Issue:


Principal Investigator

cheng yun zheng, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Department of Hematology of The 2nd Hospital of Shandong University


China: Food and Drug Administration

Study ID:




Start Date:

May 2010

Completion Date:

May 2013

Related Keywords:

  • Myelodysplastic Syndromes
  • Bone Marrow Disease
  • MDS-RA
  • Umbilical Cord/placenta-Derived MSC
  • Transplant
  • Myelodysplastic Syndromes
  • Preleukemia