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

NCT ID: NCT01129739

Last Updated: 2010-05-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-05-31

Study Completion Date

2013-05-31

Brief Summary

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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

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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.

Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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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

Group Type EXPERIMENTAL

Human umbilical cord-derived MSCs

Intervention Type OTHER

1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle

cyclosporine A (CsA)

CsA at a dose of 5 mg CsA/kg

Group Type ACTIVE_COMPARATOR

cyclosporine A (CsA)

Intervention Type OTHER

CsA 5mg/kg po for 6 months

Interventions

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Human umbilical cord-derived MSCs

1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle

Intervention Type OTHER

cyclosporine A (CsA)

CsA 5mg/kg po for 6 months

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Natural Science Foundation of China

OTHER_GOV

Sponsor Role collaborator

Shandong University

OTHER

Sponsor Role lead

Responsible Party

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Department of Hematology of the 2nd Hospital of Shandong University

Principal Investigators

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cheng yun zheng, PhD

Role: PRINCIPAL_INVESTIGATOR

Department of Hematology of The 2nd Hospital of Shandong University

Locations

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Department of Hematology of the 2nd Hospital of Shandong University

Jinan, Shandong, China

Site Status RECRUITING

Countries

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China

Central Contacts

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chengyun zheng, PhD

Role: CONTACT

+86-531-85875635

Facility Contacts

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Chengyun Zheng, PhD

Role: primary

+86-531-85875635

Other Identifiers

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No. 30670903

Identifier Type: OTHER

Identifier Source: secondary_id

CZheng

Identifier Type: -

Identifier Source: org_study_id

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