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

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ClinicalTrials.gov Identifier: NCT01129739
Recruitment Status : Unknown
Verified May 2010 by Shandong University.
Recruitment status was:  Recruiting
First Posted : May 25, 2010
Last Update Posted : May 25, 2010
Sponsor:
Collaborator:
National Natural Science Foundation of China
Information provided by:
Shandong University

Tracking Information
First Submitted Date  ICMJE May 17, 2010
First Posted Date  ICMJE May 25, 2010
Last Update Posted Date May 25, 2010
Study Start Date  ICMJE May 2010
Estimated Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 24, 2010)
  • MDS clinical symptoms (mainly anemia symptoms) [ Time Frame: 1 year ]
    Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.
  • A routine blood test [ Time Frame: 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.
  • Bone borrow cytomorphologic examination [ Time Frame: 1 year ]
    Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: May 24, 2010)
Percentage of T regulatory cell population in peripheral blood [ Time Frame: 1 year ]
Percentage of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Myelodysplastic Syndromes
Official Title  ICMJE Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat RA and RARS of MDS
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 Type  ICMJE Interventional
Study Phase Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Myelodysplastic Syndromes
Intervention  ICMJE
  • Other: Human umbilical cord-derived MSCs
    1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle
  • Other: cyclosporine A (CsA)
    CsA 5mg/kg po for 6 months
Study Arms
  • Experimental: 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
    Intervention: Other: Human umbilical cord-derived MSCs
  • Active Comparator: cyclosporine A (CsA)
    CsA at a dose of 5 mg CsA/kg
    Intervention: Other: cyclosporine A (CsA)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: May 24, 2010)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date May 2013
Estimated Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01129739
Other Study ID Numbers  ICMJE CZheng
No. 30670903 ( Other Identifier: National Natural Science Foundation of China )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Chengyun Zheng, Department of Hematology of the 2nd Hospital of Shandong University
Study Sponsor  ICMJE Shandong University
Collaborators  ICMJE National Natural Science Foundation of China
Investigators  ICMJE
Principal Investigator: cheng yun zheng, PhD Department of Hematology of The 2nd Hospital of Shandong University
PRS Account Shandong University
Verification Date May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP