Hematopoietic Stem Cell TransplantationPhase 2NCT01763099
Mesenchymal Stem Cells Combined With Cord Blood for Treatment of Graft Failure
A Phase 2 study testing Mesenchymal stem cells and Mesenchymal stem cells and cord blood in people with Hematopoietic Stem Cell Transplantation and Mesenchymal Stem Cells, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
The purpose of this study is to evaluate the utility of treating patients experiencing graft failure after autologous hematopoietic stem cell transplantation with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors or mesenchymal stem cells combined with cord blood. Read more about this study
- Who is it for?
- Ages 14 to 65 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Umbilical Cord Blood
- What is being tested?
- Mesenchymal stem cells and Mesenchymal stem cells and cord blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- Jan 2013to Jan 2016
- 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 14 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A patient age of 14-65 years
- Graft failure developing after auto-HSCT
- Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study
Who cannot join
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology,Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Ren Lin, MD, +86-020-61641613, lansinglinren@hotmail.com
About this study
Official title: Mesenchymal Stem Cells Combined With Cord Blood for Treatment of Graft Failure Following Autologous Hematopoietic Stem Cell Transplantation
The purpose of this study is to evaluate the utility of treating patients experiencing graft failure after autologous hematopoietic stem cell transplantation with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors or mesenchymal stem cells combined with cord blood. The first objective of this study was to evaluate the effect of such treatment on graft failure, and second object was to investigate the safety of such treatment.
Read the detailed description
Autologous hematopoietic stem cell transplantation (auto-HSCT) are considered the standard of care for many malignancies, such as lymphoma, myeloma and some leukemias, and so on. Graft failure after auto-HSCT is a formidable complication. It occurs in 2-9.5% of patients and is associated with considerable morbidity and mortality related to infections and hemorrhagic complications. There are various options for the management of graft failure. The most common treatment of graft failure is growth factors such as granulocyte colony-stimulating factor (G-CSF)and recombinant erythropoietin,but it usually effective in the short term and no effect on platelet counts.
Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation and so on.As an important source of hematopoietic stem cell, cord blood has been widely used in clinical practice. It is reported that cord blood combined with MSCs can increase engraftment after allogeneic hematopoietic stem cell transplantation. However, to our knowledge, the report about efficacy of treatment of graft that develops after auto-HSCT using expanded BM-derived MSCs from a third-party donor combined with cord blood is absent.If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent.
In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors or MSCs combined with cord blood in treating patients with graft failure after auto-HSCT.
- Conditions
- Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Umbilical Cord Blood, Graft Failure, Hematological Diseases
- Being tested
- Mesenchymal stem cells, Mesenchymal stem cells and cord blood
- Keywords
- Autologous Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Cord Blood, Graft failure
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mesenchymal stem cellsBiological
Mesenchymal stem cells will be intravenously infused via a central venous catheter(at a dose of 1×10^6 cells/kg, over 15 min) in day 1 and day 15 of the cycle. If the NEU and PLT levels do not attain the completely response(CR)standards after this cycle, mesenchymal stem cells combined with cord blood will be given. If the NEU and PLT levels attain the completely response(CR)or partly response(PR) standards after this cycle, another cycle with the same strategy will be given.
Mesenchymal stem cells and cord bloodBiological
Mesenchymal stem cells will be given (at a dose of 1×10^6 cells/kg,intravenously infused via a central venous catheter)on day 1.day 15, day 29 and day 43 of the cycle. Cord blood will be given intravenously infused via a central venous catheter on day 2 of the cycle.
| Study group | Role | What they receive |
|---|---|---|
| Mesenchymal stem cells | Experimental | Mesenchymal stem cells group refers to treatment with mesenchymal stem cells (1×10^6 cells/kg, intravenously) Receives: Biological: Mesenchymal stem cells |
| Mesenchymal stem cells and cord blood | Experimental | Mesenchymal stem cells and cord blood group refers to treatment with mesenchymal stem cells (at a dose of 1×10^6 cells/kg) and cord blood Receives: Biological: Mesenchymal stem cells and cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
hematopoietic recovery
Hematopoietic reconstitution post-transplantation is defined as reconstitution of both neutrophil and platelet numbers. Neutrophil reconstitution is defined as occurring on the first 3 consecutive days with an neutrophil(NEU)>0.5×10^9/L, and platelet (PLT) reconstitution is defined as the first >20×10^9/L for 3 consecutive days.
Measured: 1 year
Secondary outcomes (1)
infections, primary underlying disease relapse and any toxic side effects of MSCs treatment
Infections will be mainly focused within the first 100 days after MSCs treatment. Toxic side effects of treatment includes acute toxicity and late side effects. Acute toxicity principally involves the heart,live and kidney. Late toxic side effects involves principally the development of secondary tumors and relapse of the primary disease.
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 posted8 Jan 2013 (estimated)
- Study startJan 2013
- Primary completionJan 2015 (estimated)
- Study completionJan 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic Stem Cell TransplantationStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical UniversityThe sponsor's other registered studies.
- 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
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University People's Hospital, Sun Yat-sen University, Guangdong Provincial People's Hospital, Guangzhou General Hospital of Guangzhou Military Command, Southern Medical University, China, Third Affiliated Hospital, Sun Yat-Sen University, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou First People's Hospital, Zhongshan People's Hospital, Guangdong, China
- Responsible party
- Sponsor
- Investigators
- Qifa Liu, MD, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01763099
- Sponsor's protocol ID
- NFH-MSC-auto-HSCT-2013
- Status last verified
- Jan 2013
- First submitted
- 5 Jan 2013
- Last updated
- 16 Jan 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Koc ON, Lazarus HM. Mesenchymal stem cells: heading into the clinic. Bone Marrow Transplant. 2001 Feb;27(3):235-9. doi: 10.1038/sj.bmt.1702791.
PubMed 11277170Smith AR, Wagner JE. Alternative haematopoietic stem cell sources for transplantation: place of umbilical cord blood. Br J Haematol. 2009 Oct;147(2):246-61. doi: 10.1111/j.1365-2141.2009.07828.x.
PubMed 19796274
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stem Cells Combined With Cord Blood for Treatment of Graft Failure Following Autologous Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01763099
- Acronym / sponsor ID
- NFH-MSC-auto-HSCT-2013
- Conditions
- Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Umbilical Cord Blood, Graft Failure, Hematological Diseases
- Keywords
- Autologous Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Cord Blood, Graft failure
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- Peking University People's Hospital, Sun Yat-sen University, Guangdong Provincial People's Hospital, Guangzhou General Hospital of Guangzhou Military Command, Southern Medical University, China, Third Affiliated Hospital, Sun Yat-Sen University, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou First People's Hospital, Zhongshan People's Hospital, Guangdong, China
- Study start
- Jan 2013
- Primary completion
- Jan 2015 (estimated)
- Study completion
- Jan 2016 (estimated)
- First posted
- 8 Jan 2013 (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 utility of treating patients experiencing graft failure after autologous hematopoietic stem cell transplantation with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors or mesenchymal stem cells combined with cord blood. The first objective of this study was to evaluate the effect of such treatment on graft failure, and second object was to investigate the safety of such treatment.
Detailed description
Autologous hematopoietic stem cell transplantation (auto-HSCT) are considered the standard of care for many malignancies, such as lymphoma, myeloma and some leukemias, and so on. Graft failure after auto-HSCT is a formidable complication. It occurs in 2-9.5% of patients and is associated with considerable morbidity and mortality related to infections and hemorrhagic complications. There are various options for the management of graft failure. The most common treatment of graft failure is growth factors such as granulocyte colony-stimulating factor (G-CSF)and recombinant erythropoietin,but it usually effective in the short term and no effect on platelet counts.
Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation and so on.As an important source of hematopoietic stem cell, cord blood has been widely used in clinical practice. It is reported that cord blood combined with MSCs can increase engraftment after allogeneic hematopoietic stem cell transplantation. However, to our knowledge, the report about efficacy of treatment of graft that develops after auto-HSCT using expanded BM-derived MSCs from a third-party donor combined with cord blood is absent.If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent.
In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors or MSCs combined with cord blood in treating patients with graft failure after auto-HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mesenchymal stem cells | Experimental | Biological: Mesenchymal stem cellsMesenchymal stem cells group refers to treatment with mesenchymal stem cells (1×10^6 cells/kg, intravenously) |
| Mesenchymal stem cells and cord blood | Experimental | Biological: Mesenchymal stem cells and cord bloodMesenchymal stem cells and cord blood group refers to treatment with mesenchymal stem cells (at a dose of 1×10^6 cells/kg) and cord blood |
Who may qualify
- Ages
- 14 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- A patient age of 14-65 years
- Graft failure developing after auto-HSCT
- Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study
Key exclusion criteria (2 listed)
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
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 |
|---|---|---|
| Mesenchymal stem cellsMesenchymal stem cells will be intravenously infused via a central venous catheter(at a dose of 1×10^6 cells/kg, over 15 min) in day 1 and day 15 of the cycle. If the NEU and PLT levels do not attain the completely response(CR)standards after this cycle, mesenchymal stem cells combined with cord blood will be given. If the NEU and PLT levels attain the completely response(CR)or partly response(PR) standards after this cycle, another cycle with the same strategy will be given. | Biological | Mesenchymal stem cells |
| Mesenchymal stem cells and cord bloodMesenchymal stem cells will be given (at a dose of 1×10^6 cells/kg,intravenously infused via a central venous catheter)on day 1.day 15, day 29 and day 43 of the cycle. Cord blood will be given intravenously infused via a central venous catheter on day 2 of the cycle. | Biological | Mesenchymal stem cells and cord blood |
Primary outcome
hematopoietic recovery
Time frame: 1 year
Hematopoietic reconstitution post-transplantation is defined as reconstitution of both neutrophil and platelet numbers. Neutrophil reconstitution is defined as occurring on the first 3 consecutive days with an neutrophil(NEU)>0.5×10^9/L, and platelet (PLT) reconstitution is defined as the first >20×10^9/L for 3 consecutive days.
1 secondary outcome
infections, primary underlying disease relapse and any toxic side effects of MSCs treatment
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 16 Jan 2013.
- Background: PubMed 11277170Koc ON, Lazarus HM. Mesenchymal stem cells: heading into the clinic. Bone Marrow Transplant. 2001 Feb;27(3):235-9. doi: 10.1038/sj.bmt.1702791.
- Background: PubMed 19796274Smith AR, Wagner JE. Alternative haematopoietic stem cell sources for transplantation: place of umbilical cord blood. Br J Haematol. 2009 Oct;147(2):246-61. doi: 10.1111/j.1365-2141.2009.07828.x.
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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.
Qifa Liu, MD principal investigator
Nanfang Hospital, Southern Medical 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 Mesenchymal stem cells and Mesenchymal stem cells and cord blood in people with Hematopoietic Stem Cell Transplantation and Mesenchymal Stem Cells, run by Nanfang Hospital, Southern Medical 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 3 inclusion and 2 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 16 Jan 2013. 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 Ren Lin, MD (+86-020-61641613, lansinglinren@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
hematopoietic recovery (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01763099, which also lists 2 linked publications.
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 16 Jan 2013.