Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia

NCT ID: NCT01182662

Last Updated: 2010-08-31

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

2010-08-31

Study Completion Date

2013-08-31

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 in subject for the therapy of severe aplastic anemia (SAA).

Detailed Description

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Severe aplastic anemia (SAA) is a condition that involves a low level of red blood cells, white blood cells, and platelets without evidence of another bone marrow disease. Patients with severe aplastic anemia produce too few blood cells, causing fatigue, easy bruising and bleeding, and susceptibility to infections. In many cases, the very low blood counts result from an autoimmune process. The patient's own immune system damages their stem cells in bone marrow.

Although immune-suppressing drugs, such as corticosteroids, CsA and ATG, have been used in the treatment of SAA, however, many studies have indicated that the overall response rate to these drugs is less than 60%. Addition, the severe side effects of these immune-suppressing drugs have also been observed. The management of SAA patients therefore remains unsatisfactory and targeted therapies are needed. Human MSCs isolated from human 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 transplantation in the SAA patients.

This study will last 2 to 3 years. Participants will be randomly assigned to receive either MSC transplant and CsA therapy (experimental group) or CsA therapy alone (control group). Patients will undergo MSC transplant at the start of the study on Day 0. After 3 months, patients will receive the second MSC transplantation. After six and twelve months from the first transplantation, patients will be evaluated.

Conditions

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Aplastic Anemia

Keywords

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Bone Marrow Disease Aplastic Anemia Umbilical Cord/placenta-Derived MSC Transplantation

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 and cyclosporin

Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle and CsA 5mg/kg po for 12 months

Group Type EXPERIMENTAL

Human umbilical cord-derived MSCs and cyclosporin A

Intervention Type OTHER

1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months

cyclosporine A

cyclosporine A at a dose of 5 mg CsA/kg

Group Type ACTIVE_COMPARATOR

cyclosporin A

Intervention Type OTHER

cyclosporin A 5mg/kg po for 12 months

Interventions

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Human umbilical cord-derived MSCs and cyclosporin A

1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months

Intervention Type OTHER

cyclosporin A

cyclosporin A 5mg/kg po for 12 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. Standard of diagnosis of aplastic anemia is according to Chinese domestic classification of AA for 1987.
3. Patients must have an ECOG 0\~2.
4. No moderate or sever organ dysfunction: Ejection fraction\>45%; Creatinine \<176 umol/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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chengyun zheng, Ph. D

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, Ph. D

Role: CONTACT

Phone: +86-531-85875635

Email: [email protected]

Facility Contacts

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chengyun zheng, Ph. D

Role: primary

Other Identifiers

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No. 30670903

Identifier Type: OTHER

Identifier Source: secondary_id

kongdx

Identifier Type: -

Identifier Source: org_study_id