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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2010 by Shandong University.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
National Natural Science Foundation of China
Information provided by:
Shandong University
ClinicalTrials.gov Identifier:
NCT01129739
First received: May 17, 2010
Last updated: May 24, 2010
Last verified: May 2010
  Purpose

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).


Condition Intervention Phase
Myelodysplastic Syndromes
Other: Human umbilical cord-derived MSCs
Other: cyclosporine A (CsA)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat RA and RARS of MDS

Resource links provided by NLM:


Further study details as provided by Shandong University:

Primary Outcome Measures:
  • MDS clinical symptoms (mainly anemia symptoms) [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.

  • A routine blood test [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.


Secondary Outcome Measures:
  • Percentage of T regulatory cell population in peripheral blood [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Percentage of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.


Estimated Enrollment: 30
Study Start Date: May 2010
Estimated Study Completion Date: May 2013
Estimated Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Other: Human umbilical cord-derived MSCs
1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle
Active Comparator: cyclosporine A (CsA)
CsA at a dose of 5 mg CsA/kg
Other: cyclosporine A (CsA)
CsA 5mg/kg po for 6 months

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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01129739

Contacts
Contact: chengyun zheng, PhD +86-531-85875635 chengyun.zheng@ki.se

Locations
China, Shandong
Department of Hematology of the 2nd Hospital of Shandong University Recruiting
Jinan, Shandong, China, 250033
Contact: Chengyun Zheng, PhD    +86-531-85875635    chengyun.zheng@ki.se   
Principal Investigator: chengyun zheng, PhD         
Sponsors and Collaborators
Shandong University
National Natural Science Foundation of China
Investigators
Principal Investigator: cheng yun zheng, PhD Department of Hematology of The 2nd Hospital of Shandong University
  More Information

No publications provided

Responsible Party: Chengyun Zheng, Department of Hematology of the 2nd Hospital of Shandong University
ClinicalTrials.gov Identifier: NCT01129739     History of Changes
Other Study ID Numbers: CZheng, No. 30670903
Study First Received: May 17, 2010
Last Updated: May 24, 2010
Health Authority: China: Food and Drug Administration

Keywords provided by Shandong University:
Bone Marrow Disease
MDS-RA
MDS-RARS
Umbilical Cord/placenta-Derived MSC
Transplant

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Disease
Pathologic Processes
Cyclosporins
Cyclosporine
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Therapeutic Uses
Dermatologic Agents
Antirheumatic Agents

ClinicalTrials.gov processed this record on September 18, 2014