Clinical trial NCT01182662: Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Aplastic Anemia
Keywords: Bone Marrow Disease
Aplastic Anemia
Umbilical Cord/placenta-Derived MSC
Transplantation
Lead sponsor: Shandong University
Sponsor class: OTHER
Collaborators: name: National Natural Science Foundation of China
First posted: 2010-08-17
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

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 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.

Primary outcomes:
measure: SAA clinical symptoms
timeFrame: 1 year
measure: The number of blood cells
timeFrame: 1 year
measure: Bone borrow hemocytology
timeFrame: 1 year
Secondary outcomes:
measure: Percentage of systemic T regulatory cell population and T lymphocyte subsets
timeFrame: 1 year
Other outcomes:
Locations:
city: Jinan
country: China
facility: Department of Hematology of the 2nd Hospital of Shandong University
state: Shandong
Study officials:
affiliation: Department of Hematology of The 2nd Hospital of Shandong University
name: chengyun zheng, Ph. D
role: PRINCIPAL_INVESTIGATOR
References: