Aplastic AnemiaPhase 2NCT01182662
Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia
A Phase 2 study testing Human umbilical cord-derived MSCs and cyclosporin A and cyclosporin A in people with Aplastic Anemia, run by Shandong University at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Human umbilical cord-derived MSCs and cyclosporin A and cyclosporin A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- Aug 2010to Aug 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Psychiatric condition that would limit informed consent.
- HIV positive
- Positive Pregnancy Test
- Patient has enrolled another clinical trial study within last 4 weeks.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology of the 2nd Hospital of Shandong University
Jinan, Shandong, 250033
chengyun zheng, Ph. D, +86-531-85875635, chengyun.zheng@ki.se
About this study
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA
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).
Read the 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.
- Conditions
- Aplastic Anemia
- Being tested
- Human umbilical cord-derived MSCs and cyclosporin A, cyclosporin A
- Keywords
- Bone Marrow Disease, Aplastic Anemia, Umbilical Cord/placenta-Derived MSC, Transplantation
- Medical subject headings
- Anemia, Aplastic, Bone Marrow Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human umbilical cord-derived MSCs and cyclosporin AOther
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
cyclosporin AOther
cyclosporin A 5mg/kg po for 12 months
| Study group | Role | What they receive |
|---|---|---|
| Human umbilical cord-derived MSCs and cyclosporin | Experimental | 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 Receives: Other: Human umbilical cord-derived MSCs and cyclosporin A |
| cyclosporine A | Active comparator | cyclosporine A at a dose of 5 mg CsA/kg Receives: Other: cyclosporin A |
What is being measured
The primary outcome is the main question the study is designed to answer.
SAA clinical symptoms
Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
Measured: 1 year
The number of blood cells
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
Measured: 1 year
Bone borrow hemocytology
Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.
Measured: 1 year
Secondary outcomes (1)
Percentage of systemic T regulatory cell population and T lymphocyte subsets
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2010 (estimated)
- Study startAug 2010
- Primary completionAug 2013 (estimated)
- Study completionAug 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong University
- Sponsor type
- University, hospital or other
- Collaborators
- National Natural Science Foundation of China
- Investigators
- chengyun zheng, Ph. D, Principal investigator, Department of Hematology of The 2nd Hospital of Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01182662
- Sponsor's protocol ID
- kongdx
- Other IDs
- No. 30670903
- Status last verified
- Aug 2010
- First submitted
- 11 Aug 2010
- Last updated
- 31 Aug 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA
- ClinicalTrials.gov ID
- NCT01182662
- Acronym / sponsor ID
- kongdx
- Conditions
- Aplastic Anemia
- Keywords
- Bone Marrow Disease, Aplastic Anemia, Umbilical Cord/placenta-Derived MSC, Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Shandong UniversityUniversity, hospital or other
- Collaborators
- National Natural Science Foundation of China
- Study start
- Aug 2010
- Primary completion
- Aug 2013 (estimated)
- Study completion
- Aug 2013 (estimated)
- First posted
- 17 Aug 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Human umbilical cord-derived MSCs and cyclosporin | Experimental | Other: Human umbilical cord-derived MSCs and cyclosporin AHuman 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 |
| cyclosporine A | Active comparator | Other: cyclosporin Acyclosporine A at a dose of 5 mg CsA/kg |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (4 listed)
- Psychiatric condition that would limit informed consent.
- HIV positive
- Positive Pregnancy Test
- Patient has enrolled another clinical trial study within last 4 weeks.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human umbilical cord-derived MSCs and cyclosporin A1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months | Other | Human umbilical cord-derived MSCs and cyclosporin |
| cyclosporin Acyclosporin A 5mg/kg po for 12 months | Other | cyclosporine A |
Primary outcome
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.
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
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 secondary outcome
Percentage of systemic T regulatory cell population and T lymphocyte subsets
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2010.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
chengyun zheng, Ph. D principal investigator
Department of Hematology of The 2nd Hospital of Shandong University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Human umbilical cord-derived MSCs and cyclosporin A and cyclosporin A in people with Aplastic Anemia, run by Shandong University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 31 Aug 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact chengyun zheng, Ph. D (+86-531-85875635, chengyun.zheng@ki.se), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
SAA clinical symptoms (time frame: 1 year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2010.