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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2010 by Shandong University.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
National Natural Science Foundation of China
Information provided by:
Shandong University
ClinicalTrials.gov Identifier:
NCT01182662
First received: August 11, 2010
Last updated: August 30, 2010
Last verified: August 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 in subject for the therapy of severe aplastic anemia (SAA).


Condition Intervention Phase
Aplastic Anemia
Other: Human umbilical cord-derived MSCs and cyclosporin A
Other: cyclosporin A
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 SAA

Resource links provided by NLM:


Further study details as provided by Shandong University:

Primary Outcome Measures:
  • SAA clinical symptoms [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.

  • The number of blood cells [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    The number of blood cells, which contains WBC, Neu, RBC, Hb,PLT and reticulocyte, will be mainly tested monthly after transplantion of MSCs for one year

  • Bone borrow hemocytology [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.


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


Estimated Enrollment: 30
Study Start Date: August 2010
Estimated Study Completion Date: August 2013
Estimated Primary Completion Date: August 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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
Other: 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
Active Comparator: cyclosporine A
cyclosporine A at a dose of 5 mg CsA/kg
Other: cyclosporin A
cyclosporin A 5mg/kg po for 12 months

Detailed Description:

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.

  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. 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.
  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: NCT01182662

Contacts
Contact: chengyun zheng, Ph. D +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, Ph. D    +86-531-85875635    chengyun.zheng@ki.se   
Sponsors and Collaborators
Shandong University
National Natural Science Foundation of China
Investigators
Principal Investigator: chengyun zheng, Ph. D 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: NCT01182662     History of Changes
Other Study ID Numbers: kongdx, No. 30670903
Study First Received: August 11, 2010
Last Updated: August 30, 2010
Health Authority: China: Food and Drug Administration

Keywords provided by Shandong University:
Bone Marrow Disease
Aplastic Anemia
Umbilical Cord/placenta-Derived MSC
Transplantation

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Bone Marrow Diseases
Hematologic Diseases
Cyclosporine
Cyclosporins
Anti-Infective Agents
Antifungal Agents
Antirheumatic Agents
Dermatologic Agents
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014