- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01182662
Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia
Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Anticipated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: chengyun zheng, Ph. D
- Phone Number: +86-531-85875635
- Email: chengyun.zheng@ki.se
Study Locations
-
-
Shandong
-
Jinan, Shandong, China, 250033
- Recruiting
- Department of Hematology of the 2nd Hospital of Shandong University
-
Contact:
- chengyun zheng, Ph. D
- Phone Number: +86-531-85875635
- Email: chengyun.zheng@ki.se
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patient age 18~80 years old with plan to infuse MSCs.
- Standard of diagnosis of aplastic anemia is according to Chinese domestic classification of AA for 1987.
- Patients must have an ECOG 0~2.
- No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.
- No active severe viral or fungus infection.
- Each patient must sign written informed consent.
Exclusion Criteria:
- Psychiatric condition that would limit informed consent.
- HIV positive
- Positive Pregnancy Test
- Patient has enrolled another clinical trial study within last 4 weeks.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Single
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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
|
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
|
cyclosporin A 5mg/kg po for 12 months
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
SAA clinical symptoms
Time Frame: 1 year
|
Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
|
1 year
|
The number of blood cells
Time Frame: 1 year
|
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
|
1 year
|
Bone borrow hemocytology
Time Frame: 1 year
|
Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of systemic T regulatory cell population and T lymphocyte subsets
Time Frame: 1 year
|
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.
|
1 year
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: chengyun zheng, Ph. D, Department of Hematology of the 2nd Hospital of Shandong University
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Bone Marrow Diseases
- Hematologic Diseases
- Bone Marrow Failure Disorders
- Anemia
- Anemia, Aplastic
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Enzyme Inhibitors
- Antirheumatic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Dermatologic Agents
- Antifungal Agents
- Calcineurin Inhibitors
- Cyclosporine
- Cyclosporins
Other Study ID Numbers
- kongdx
- No. 30670903 (Other Identifier: National Natural Science Foundation of China)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Aplastic Anemia
-
Assistance Publique - Hôpitaux de ParisNot yet recruitingSevere Aplastic Anemia | Idiopathic Aplastic Anemia | Moderate Aplastic Anemia Requiring Transfusions
-
University of UtahNovartisCompletedSevere Aplastic Anemia | Moderate Aplastic Anemia | Very Severe Aplastic AnemiaUnited States
-
City of Hope Medical CenterNational Cancer Institute (NCI)RecruitingRecurrent Severe Aplastic Anemia | Refractory Severe Aplastic AnemiaUnited States
-
Peking University People's HospitalRecruiting
-
Boston Children's HospitalNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaboratorsRecruitingSevere Aplastic AnemiaUnited States
-
Federal Research Institute of Pediatric Hematology...RecruitingAcquired Aplastic AnemiaRussian Federation
-
Shanghai General Hospital, Shanghai Jiao Tong University...Ruijin Hospital; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine and other collaboratorsCompleted
-
Nagoya UniversityUnknownAcquired Aplastic Anemia.Japan
-
Navy General Hospital, BeijingPeking Union Medical College Hospital; Cancer Institute and Hospital, Chinese... and other collaboratorsUnknownSevere Aplastic AnemiaChina
-
Jiangsu HengRui Medicine Co., Ltd.Recruiting
Clinical Trials on Human umbilical cord-derived MSCs and cyclosporin A
-
Institute of Hematology & Blood Diseases HospitalActive, not recruitingThrombocytopenia | Mesenchymal Stem CellsChina
-
University of JordanActive, not recruiting
-
Hai LiNot yet recruiting
-
Cytopeutics Sdn. Bhd.Universiti Kebangsaan Malaysia Medical CentreCompleted
-
Gangnam Severance HospitalDaewoong Pharmaceutical Co. LTD.CompletedRecessive Dystrophic Epidermolysis BullosaKorea, Republic of
-
Affiliated Hospital to Academy of Military Medical...Unknown
-
Cell Energy Life Sciences Group Co. LTDBeijing YouAn Hospital; Beijing 302 Hospital; Shenzhen Third People's Hospital; Fifth Hospital of Shijiazhuang CityRecruiting
-
Limin RongCompleted
-
The Affiliated Nanjing Drum Tower Hospital of Nanjing...UnknownSystemic Lupus ErythematosusChina
-
Second Affiliated Hospital, School of Medicine,...Recruiting