Myelodysplastic SyndromesPhase 2NCT01129739
Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Myelodysplastic Syndromes
A Phase 2 study testing Human umbilical cord-derived MSCs and cyclosporine A (CsA) in people with Myelodysplastic Syndromes, 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 on the subjects for refractory anemia (RA) and refractory anemia with ring sideroblast (RARS) of myelodysplastic syndromes (MDS). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Human umbilical cord-derived MSCs and cyclosporine A (CsA)
- 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
- May 2010to May 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.
- Histologically documented or cytologically confirmed diagnosis of MDS with WHO classification of MDS-RA and MDS-RARS.
- Patients must have an ECOG 0~2.
- No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 mmol/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, PhD, +86-531-85875635, chengyun.zheng@ki.se
chengyun zheng, PhD
About this study
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat RA and RARS of MDS
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 on the subjects for refractory anemia (RA) and refractory anemia with ring sideroblast (RARS) of myelodysplastic syndromes (MDS).
Read the detailed description
Myelodysplastic syndromes are bone marrow stem cell disorders resulting in disorderly and ineffective hematopoiesis. MDS is characterized by variable degrees of cytopenias (anemia, neutropenia, and thrombocytopenia ) and risk of transformation to leukemia.
To date treatment of MDS is unsatisfactory: chemotherapy has a limited role in the management of leukemic progression; autologous stem cell transplantation does not prolong relapse-free survival and stem cell transplantation is poorly tolerated in older individuals. Some MDS patients have been shown to respond to a wide variety of immunosuppressive agents ranging from corticosteroids to CsA and antithymocyte globulin (ATG). However, the overall response rate is less than 30%. In fact, few treatments appear to change the natural history of MDS.
The management of MDS patients therefore remains to be improved. Human MSCs isolated from Wharton's jelly of the 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 transplant in the MDS patients.
This study will last about 3 years. Participants will be randomly assigned to receive either MSC transplant (Group 1) or CsA therapy alone (Group 2). Patients will undergo MSC transplant at the start of the study (defined as Day 0). After 3 months, patients will receive the second MSC transplantation when one responds well to the treatment. After 3, 6 and 12 months from the first transplantation, patients will be evaluated.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Human umbilical cord-derived MSCs, cyclosporine A (CsA)
- Keywords
- Bone Marrow Disease, MDS-RA, MDS-RARS, Umbilical Cord/placenta-Derived MSC, Transplant
- Medical subject headings
- Myelodysplastic Syndromes, 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 MSCsOther
1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle
cyclosporine A (CsA)Other
CsA 5mg/kg po for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Human umbilical cord-derived MSCs | Experimental | Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle Receives: Other: Human umbilical cord-derived MSCs |
| cyclosporine A (CsA) | Active comparator | CsA at a dose of 5 mg CsA/kg Receives: Other: cyclosporine A (CsA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
MDS clinical symptoms (mainly anemia symptoms)
Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.
Measured: 1 year
A routine blood test
A routine blood test, which contains WBC, Neu, RBC, Hb and PLT, will be mainly tested in every month after transplanting MSCs for one year.
Measured: 1 year
Bone borrow cytomorphologic examination
Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.
Measured: 1 year
Secondary outcomes (1)
Percentage of T regulatory cell population in peripheral blood
Percentage of T regulatory cell population 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 posted25 May 2010 (estimated)
- Study startMay 2010
- Primary completionMay 2013 (estimated)
- Study completionMay 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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
- cheng yun zheng, PhD, Principal investigator, Department of Hematology of The 2nd Hospital of Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01129739
- Sponsor's protocol ID
- CZheng
- Other IDs
- No. 30670903
- Status last verified
- May 2010
- First submitted
- 17 May 2010
- Last updated
- 25 May 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 May 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 RA and RARS of MDS
- ClinicalTrials.gov ID
- NCT01129739
- Acronym / sponsor ID
- CZheng
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Bone Marrow Disease, MDS-RA, MDS-RARS, Umbilical Cord/placenta-Derived MSC, Transplant
- 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
- May 2010
- Primary completion
- May 2013 (estimated)
- Study completion
- May 2013 (estimated)
- First posted
- 25 May 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 on the subjects for refractory anemia (RA) and refractory anemia with ring sideroblast (RARS) of myelodysplastic syndromes (MDS).
Detailed description
Myelodysplastic syndromes are bone marrow stem cell disorders resulting in disorderly and ineffective hematopoiesis. MDS is characterized by variable degrees of cytopenias (anemia, neutropenia, and thrombocytopenia ) and risk of transformation to leukemia.
To date treatment of MDS is unsatisfactory: chemotherapy has a limited role in the management of leukemic progression; autologous stem cell transplantation does not prolong relapse-free survival and stem cell transplantation is poorly tolerated in older individuals. Some MDS patients have been shown to respond to a wide variety of immunosuppressive agents ranging from corticosteroids to CsA and antithymocyte globulin (ATG). However, the overall response rate is less than 30%. In fact, few treatments appear to change the natural history of MDS.
The management of MDS patients therefore remains to be improved. Human MSCs isolated from Wharton's jelly of the 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 transplant in the MDS patients.
This study will last about 3 years. Participants will be randomly assigned to receive either MSC transplant (Group 1) or CsA therapy alone (Group 2). Patients will undergo MSC transplant at the start of the study (defined as Day 0). After 3 months, patients will receive the second MSC transplantation when one responds well to the treatment. After 3, 6 and 12 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 | Experimental | Other: Human umbilical cord-derived MSCsHuman umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle |
| cyclosporine A (CsA) | Active comparator | Other: cyclosporine A (CsA)CsA 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.
- Histologically documented or cytologically confirmed diagnosis of MDS with WHO classification of MDS-RA and MDS-RARS.
- Patients must have an ECOG 0~2.
- No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 mmol/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 MSCs1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle | Other | Human umbilical cord-derived MSCs |
| cyclosporine A (CsA)CsA 5mg/kg po for 6 months | Other | cyclosporine A (CsA) |
Primary outcome
MDS clinical symptoms (mainly anemia symptoms)
Time frame: 1 year
Anemia symptoms will be mainly observed in every week after transplanting MSCs for one year.
A routine blood test
Time frame: 1 year
A routine blood test, which contains WBC, Neu, RBC, Hb and PLT, will be mainly tested in every month after transplanting MSCs for one year.
Bone borrow cytomorphologic examination
Time frame: 1 year
Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.
1 secondary outcome
Percentage of T regulatory cell population in peripheral blood
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 25 May 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.
cheng yun zheng, PhD 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 cyclosporine A (CsA) in people with Myelodysplastic Syndromes, 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 25 May 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, PhD (+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?
MDS clinical symptoms (mainly anemia 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 25 May 2010.