Severe Aplastic AnemiaPhase 2NCT02247973

Mesenchymal Stem Cells Co-transplantation in Alternative Donor Transplantation of Severe Aplastic Anemia.

A Phase 2 study testing mesenchymal stem cells in people with Severe Aplastic Anemia, run by Guangzhou General Hospital of Guangzhou Military Command at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

Search all trials

Trial summary

The study is a phase II trial designed to evaluate the efficacy and safety of co-transplantation with bone marrow derived mesenchymal stem cells from related donors in alternative donor transplantation of severe aplastic anemia. Read more about this study

Who is it for?: Ages 14 to 50 years
Conditions the study is for: Severe Aplastic Anemia
What is being tested?: mesenchymal stem cells
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 100 participants
Where: 1 siteChina
Study dates: Feb 2013to Feb 2018
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 50 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

In line with the 2009 Edition (United Kingdom) aplastic anemia diagnostic criteria for SAA or VSAA;
Age less than 50 years old,willing to transplant；
No HLA-identical sibling donor；
Have HLA-mismatched related donors or unrelated donors ( ≥5/10 HLA matched loci in related donors; ≥8/10 HLA matched loci in unrelated donors )
No serious infection or acute hemorrhage;
Cardiac ultrasound examination showed left ventricular ejection fraction is greater than 50%;

Show all 10Show fewer

Both transaminase and serum creatinine level are no more than twice times the upper limit of normal value (ULN);
No acute infectious disease;
Ability to understand and the willingness to sign a written informed consent document.
ECOG score of 0-2 points.

Who cannot join

Patients with severe infection or active bleeding;
With severe cardiac insufficiency, left ventricular ejection fraction <50%;
With severe liver dysfunction, liver function (ALT and the TBIL) is higher than the ULN 3 times;
With severe renal insufficiency, renal function (Cr) is twice higher than the ULN; or 24-hour urine creatinine clearance rate (Ccr) lower than 50ml/min;
Active tuberculosis, severe acute hepatitis and other infectious diseases in active period;
ECOG score more than 3 points;
Accompanied by malignant tumors and other clonal disease;
Poor compliance and the researchers considered unsuitable for MSC infusion.

Trial locations and contacts

1 site in 1 country.

China1 site

Guangzhou General Hospital of Guangzhou Military Command
Guangzhou, Guangdong, 510010

Other trials in China

About this study

Official title: PhaseⅡTrial of Co-transplantation With Bone Marrow Derived Mesenchymal Stem Cells From Related Donors in Alternative Donor Transplantation of Severe Aplastic Anemia.

Read the detailed description

Aplastic anemia (AA) is an autoimmune hematologic stem cell disease mediated by activated T-lymphocytes that leads to bone marrow dysfunction. In the presence of an empty marrow, pancytopenia, and transfusion dependence, the severity of the disease is based on neutrophil (PMN) count: nonsevere AA (nSAA; PMN > 0.5 × 109/L), severe AA (SAA;PMN 0.2- 0.5 × 109/L), and very severe AA (vSAA; PMN< 0.2 × 109/L).

Allogeneic BMT from an HLA-identical sibling donor or matched-alternative donor is the treatment of choice for patients with aplastic anaemia.Transplantation for patients with severe aplastic anaemia from an HLA identical sibling donor is now very successful with a 75-90% chance of long term cure and with overall survival of between 65% and 73% at 5 years for matched-alternative donor transplantation. However, these two approachs are limited by the availability of HLA-matched donors.

Patients without HLA-identical sibling donor or matched-alternative donor can be offered immunosuppressive treatment (IST) involving injections of Anti-thymocyte globulin (ATG) in combination with cyclosporine (CsA). The treatment response with ATG is at best between 60-80%, 30%-40% patients relapse following an initial response to treatment. Moreover, a recent study has shown that on multivariate analysis of response at 6 months, only younger age, absolute reticulocyte count (ARC) and absolute lymphocyte count (ALC), correlate with response to ATG. Patients with SAA or vSAA, with much lower ARC and ALC, were poor response to IST and have high risks of dying of infection and bleeding.

Nowadays, with advances in transplant technology, HLA-mismatched related donors and unrelated donors transplantation has achieved good clinical results. Data from the XJ Huang indicated that patients with HLA-mismatched related donors achieved 100% donor myeloid engraftment and have a survival rate of 64.6±12.4%. Retrospectively analyzed results for 154 patients with acquired SAA who received BMT from unrelated donors identified through the Japan Marrow Donor Program showed the probability of OS at 5 years was 56% (95% confidence interval, 34%-78%).

Compared with malignant disease, mismatched related donor or unrelated donor HSCT for SAA involves distinct challenges mainly associated with high graft failure and high GVHD. So, if we can find a way to promote implantation meanwhile prevent or reduce GVHD , the efficacy of HLA-mismatched related donors transplantation can improve.

Mesenchymal stem cells (MSCs) are multi-potent non-hematopoietic progenitors mainly found in BM, cord blood, and adipose tissue. MSCs are attractive because of the ease with which they can be isolated and expanded ex vivo, their ability to undergo multilineage differentiation, and their lack of immunogenicity. These cells were shown to provide support for the growth and differentiation of hematopoietic progenitor cells in BM micro-environments. In additon, preliminary studies have shown clinical effectiveness of allogeneic MSC in the treatment of refractory graft-versus-host disease and an improvement in or resolution of severe aGVHD when co-transplantation with MSCs. Due to these properties, MSCs have become an interesting candidate for use in cellular therapy and are considered "theoretically perfect cells" for potential clinical use against AA mismatched related donors transplantation.

Conditions: Severe Aplastic Anemia
Being tested: mesenchymal stem cells
Medical subject headings: Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

mesenchymal stem cellsBiological
Also called Multipotent Mesenchymal Stromal Cells
Intravenous administration of up to 1~2x10^6 MSCs per kg,for 2 times,d0 and d14
mesenchymal stem cellsBiological
bone marrow derived mesenchymal stem cells from related donors.

Study groups in NCT02247973
Study group	Role	What they receive
Mesenchymal stem cells	Experimental	Intravenous bone marrow derived mesenchymal stem cells infusion from related donor to patients with severe aplastic anemia. Receives: Biological: mesenchymal stem cells

What is being measured

The primary outcome is the main question the study is designed to answer.

survival rate
The 2-year disease-free survival and overall survival.
Measured: up to 2 years after HSCT

Secondary outcomes (5)

acute GVHD
The incidence of acute GVHD after HSCT.
Measured: UP to 3 months after HSCT
chronic GVHD
The incidence of chronic GVHD after HSCT.
Measured: UP to 2 years after HSCT
Transplant-related mortality
Measured: UP to 1 months after HSCT
Rates of relapse
Measured: UP to 2 years after HSCT
The implantation
The implantation rate and implantation time.
Measured: Up to 4 weeks after HSCT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Feb 2013Feb 2018

First posted25 Sep 2014 (estimated)
Study startFeb 2013
Primary completionFeb 2017 (estimated)
Study completionFeb 2018 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (15)Papers the sponsor has linked to this study.
Other trials for Severe 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 100 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02247973
Sponsor's protocol ID: MSC-alternative donor SCT-SAA
Status last verified: Sep 2014
First submitted: 17 Sep 2014
Last updated: 25 Sep 2014
Results posted: No
Data monitoring committee: Yes

Publications

Marsh JC, Ball SE, Cavenagh J, Darbyshire P, Dokal I, Gordon-Smith EC, Keidan J, Laurie A, Martin A, Mercieca J, Killick SB, Stewart R, Yin JA; British Committee for Standards in Haematology. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009 Oct;147(1):43-70. doi: 10.1111/j.1365-2141.2009.07842.x. Epub 2009 Aug 10. No abstract available.
PubMed 19673883
Bacigalupo A, Locatelli F, Lanino E, Marsh J, Socie G, Maury S, Prete A, Locasciulli A, Cesaro S, Passweg J; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005 Dec;36(11):947-50. doi: 10.1038/sj.bmt.1705165.
PubMed 16205733
Deeg HJ, O'Donnell M, Tolar J, Agarwal R, Harris RE, Feig SA, Territo MC, Collins RH, McSweeney PA, Copelan EA, Khan SP, Woolfrey A, Storer B. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006 Sep 1;108(5):1485-91. doi: 10.1182/blood-2006-03-005041. Epub 2006 May 9.
PubMed 16684959
George B, Mathews V, Viswabandya A, Lakshmi KM, Srivastava A, Chandy M. Allogeneic hematopoietic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia--a single-center analysis of 100 patients. Pediatr Hematol Oncol. 2010 Mar;27(2):122-31. doi: 10.3109/08880010903540542.
PubMed 20201693
Kojima S, Matsuyama T, Kato S, Kigasawa H, Kobayashi R, Kikuta A, Sakamaki H, Ikuta K, Tsuchida M, Hoshi Y, Morishima Y, Kodera Y. Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program. Blood. 2002 Aug 1;100(3):799-803. doi: 10.1182/blood.v100.3.799.
PubMed 12130489
Noort WA, Kruisselbrink AB, in't Anker PS, Kruger M, van Bezooijen RL, de Paus RA, Heemskerk MH, Lowik CW, Falkenburg JH, Willemze R, Fibbe WE. Mesenchymal stem cells promote engraftment of human umbilical cord blood-derived CD34(+) cells in NOD/SCID mice. Exp Hematol. 2002 Aug;30(8):870-8. doi: 10.1016/s0301-472x(02)00820-2.
PubMed 12160838
Le Blanc K, Rasmusson I, Sundberg B, Gotherstrom C, Hassan M, Uzunel M, Ringden O. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet. 2004 May 1;363(9419):1439-41. doi: 10.1016/S0140-6736(04)16104-7.
PubMed 15121408
Ringden O, Uzunel M, Rasmusson I, Remberger M, Sundberg B, Lonnies H, Marschall HU, Dlugosz A, Szakos A, Hassan Z, Omazic B, Aschan J, Barkholt L, Le Blanc K. Mesenchymal stem cells for treatment of therapy-resistant graft-versus-host disease. Transplantation. 2006 May 27;81(10):1390-7. doi: 10.1097/01.tp.0000214462.63943.14.
PubMed 16732175
Le Blanc K, Frassoni F, Ball L, Locatelli F, Roelofs H, Lewis I, Lanino E, Sundberg B, Bernardo ME, Remberger M, Dini G, Egeler RM, Bacigalupo A, Fibbe W, Ringden O; Developmental Committee of the European Group for Blood and Marrow Transplantation. Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet. 2008 May 10;371(9624):1579-86. doi: 10.1016/S0140-6736(08)60690-X.
PubMed 18468541
Ball LM, Bernardo ME, Roelofs H, Lankester A, Cometa A, Egeler RM, Locatelli F, Fibbe WE. Cotransplantation of ex vivo expanded mesenchymal stem cells accelerates lymphocyte recovery and may reduce the risk of graft failure in haploidentical hematopoietic stem-cell transplantation. Blood. 2007 Oct 1;110(7):2764-7. doi: 10.1182/blood-2007-04-087056. Epub 2007 Jul 16.
PubMed 17638847
Fang B, Li N, Song Y, Li J, Zhao RC, Ma Y. Cotransplantation of haploidentical mesenchymal stem cells to enhance engraftment of hematopoietic stem cells and to reduce the risk of graft failure in two children with severe aplastic anemia. Pediatr Transplant. 2009 Jun;13(4):499-502. doi: 10.1111/j.1399-3046.2008.01002.x. Epub 2008 Jul 30.
PubMed 18673358
Wang H, Wang Z, Xue M, Liu J, Yan H, Guo Z. Co-transfusion of haplo-identical hematopoietic and mesenchymal stromal cells to treat a patient with severe aplastic. Cytotherapy. 2010 Jul;12(4):563-5. doi: 10.3109/14653241003695059.
PubMed 20380540
Jaganathan BG, Tisato V, Vulliamy T, Dokal I, Marsh J, Dazzi F, Bonnet D. Effects of MSC co-injection on the reconstitution of aplastic anemia patient following hematopoietic stem cell transplantation. Leukemia. 2010 Oct;24(10):1791-5. doi: 10.1038/leu.2010.164. Epub 2010 Aug 19. No abstract available.
PubMed 20724985
Fang B, Song Y, Li N, Li J, Han Q, Zhao RC. Mesenchymal stem cells for the treatment of refractory pure red cell aplasia after major ABO-incompatible hematopoietic stem cell transplantation. Ann Hematol. 2009 Mar;88(3):261-6. doi: 10.1007/s00277-008-0599-0. Epub 2008 Sep 4.
PubMed 18769919
Xiao Y, Jiang ZJ, Pang Y, Li L, Gao Y, Xiao HW, Li YH, Zhang H, Liu Q. Efficacy and safety of mesenchymal stromal cell treatment from related donors for patients with refractory aplastic anemia. Cytotherapy. 2013 Jul;15(7):760-6. doi: 10.1016/j.jcyt.2013.03.007.
PubMed 23731760

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes, Aplastic Anemias
Active, not recruitingPhase 2
Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
Aplastic Anemia, TPO Receptor Agonists
RecruitingObservational study
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Severe Aplastic Anemia, Telomere Biology Disorders

All Severe Aplastic Anemia trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: PhaseⅡTrial of Co-transplantation With Bone Marrow Derived Mesenchymal Stem Cells From Related Donors in Alternative Donor Transplantation of Severe Aplastic Anemia.
ClinicalTrials.gov ID: NCT02247973
Acronym / sponsor ID: MSC-alternative donor SCT-SAA
Conditions: Severe Aplastic Anemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 100 participants (estimated)
Lead sponsor: Guangzhou General Hospital of Guangzhou Military CommandUniversity, hospital or other
Collaborators: Guangzhou First People's Hospital, Nanfang Hospital, Southern Medical University, Southern Medical University, China, First Affiliated Hospital, Sun Yat-Sen University, Second Affiliated Hospital, Sun Yat-Sen University, Third Affiliated Hospital, Sun Yat-Sen University, Fifth Affiliated Hospital, Sun Yat-Sen University, Guangdong Provincial People's Hospital, The Second People's Hospital of GuangDong Province, First Affiliated Hospital of Jinan University, The First Affiliated Hospital of Guangzhou Medical University, Second Affiliated Hospital of Guangzhou Medical University, Peking University Shenzhen Hospital, Shenzhen Second People's Hospital
Study start: Feb 2013
Primary completion: Feb 2017 (estimated)
Study completion: Feb 2018 (estimated)
First posted: 25 Sep 2014 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Mesenchymal stem cells	Experimental	Biological: mesenchymal stem cellsIntravenous bone marrow derived mesenchymal stem cells infusion from related donor to patients with severe aplastic anemia.

Who may qualify

Ages: 14 to 50 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (10 listed)

In line with the 2009 Edition (United Kingdom) aplastic anemia diagnostic criteria for SAA or VSAA;
Age less than 50 years old,willing to transplant；
No HLA-identical sibling donor；
Have HLA-mismatched related donors or unrelated donors ( ≥5/10 HLA matched loci in related donors; ≥8/10 HLA matched loci in unrelated donors )
No serious infection or acute hemorrhage;

Key exclusion criteria (8 listed)

Patients with severe infection or active bleeding;
With severe cardiac insufficiency, left ventricular ejection fraction <50%;
With severe liver dysfunction, liver function (ALT and the TBIL) is higher than the ULN 3 times;
With severe renal insufficiency, renal function (Cr) is twice higher than the ULN; or 24-hour urine creatinine clearance rate (Ccr) lower than 50ml/min;
Active tuberculosis, severe acute hepatitis and other infectious diseases in active period;

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 cellsAlso: Multipotent Mesenchymal Stromal CellsIntravenous administration of up to 1~2x10^6 MSCs per kg,for 2 times,d0 and d14	Biological	Mesenchymal stem cells
mesenchymal stem cellsbone marrow derived mesenchymal stem cells from related donors.	Biological	Mesenchymal stem cells

Primary outcome

survival rate
Time frame: up to 2 years after HSCT
The 2-year disease-free survival and overall survival.

5 secondary outcomes

acute GVHD
Time frame: UP to 3 months after HSCT
chronic GVHD
Time frame: UP to 2 years after HSCT
Transplant-related mortality
Time frame: UP to 1 months after HSCT
Rates of relapse
Time frame: UP to 2 years after HSCT
The implantation
Time frame: Up to 4 weeks after HSCT

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2014.
Background: PubMed 19673883Marsh JC, Ball SE, Cavenagh J, Darbyshire P, Dokal I, Gordon-Smith EC, Keidan J, Laurie A, Martin A, Mercieca J, Killick SB, Stewart R, Yin JA; British Committee for Standards in Haematology. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009 Oct;147(1):43-70. doi: 10.1111/j.1365-2141.2009.07842.x. Epub 2009 Aug 10. No abstract available.
Background: PubMed 16205733Bacigalupo A, Locatelli F, Lanino E, Marsh J, Socie G, Maury S, Prete A, Locasciulli A, Cesaro S, Passweg J; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005 Dec;36(11):947-50. doi: 10.1038/sj.bmt.1705165.
Background: PubMed 16684959Deeg HJ, O'Donnell M, Tolar J, Agarwal R, Harris RE, Feig SA, Territo MC, Collins RH, McSweeney PA, Copelan EA, Khan SP, Woolfrey A, Storer B. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006 Sep 1;108(5):1485-91. doi: 10.1182/blood-2006-03-005041. Epub 2006 May 9.
Background: PubMed 20201693George B, Mathews V, Viswabandya A, Lakshmi KM, Srivastava A, Chandy M. Allogeneic hematopoietic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia--a single-center analysis of 100 patients. Pediatr Hematol Oncol. 2010 Mar;27(2):122-31. doi: 10.3109/08880010903540542.
Background: PubMed 12130489Kojima S, Matsuyama T, Kato S, Kigasawa H, Kobayashi R, Kikuta A, Sakamaki H, Ikuta K, Tsuchida M, Hoshi Y, Morishima Y, Kodera Y. Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program. Blood. 2002 Aug 1;100(3):799-803. doi: 10.1182/blood.v100.3.799.
Background: PubMed 12160838Noort WA, Kruisselbrink AB, in't Anker PS, Kruger M, van Bezooijen RL, de Paus RA, Heemskerk MH, Lowik CW, Falkenburg JH, Willemze R, Fibbe WE. Mesenchymal stem cells promote engraftment of human umbilical cord blood-derived CD34(+) cells in NOD/SCID mice. Exp Hematol. 2002 Aug;30(8):870-8. doi: 10.1016/s0301-472x(02)00820-2.
Background: PubMed 15121408Le Blanc K, Rasmusson I, Sundberg B, Gotherstrom C, Hassan M, Uzunel M, Ringden O. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet. 2004 May 1;363(9419):1439-41. doi: 10.1016/S0140-6736(04)16104-7.
Background: PubMed 16732175Ringden O, Uzunel M, Rasmusson I, Remberger M, Sundberg B, Lonnies H, Marschall HU, Dlugosz A, Szakos A, Hassan Z, Omazic B, Aschan J, Barkholt L, Le Blanc K. Mesenchymal stem cells for treatment of therapy-resistant graft-versus-host disease. Transplantation. 2006 May 27;81(10):1390-7. doi: 10.1097/01.tp.0000214462.63943.14.
Background: PubMed 18468541Le Blanc K, Frassoni F, Ball L, Locatelli F, Roelofs H, Lewis I, Lanino E, Sundberg B, Bernardo ME, Remberger M, Dini G, Egeler RM, Bacigalupo A, Fibbe W, Ringden O; Developmental Committee of the European Group for Blood and Marrow Transplantation. Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet. 2008 May 10;371(9624):1579-86. doi: 10.1016/S0140-6736(08)60690-X.
Background: PubMed 17638847Ball LM, Bernardo ME, Roelofs H, Lankester A, Cometa A, Egeler RM, Locatelli F, Fibbe WE. Cotransplantation of ex vivo expanded mesenchymal stem cells accelerates lymphocyte recovery and may reduce the risk of graft failure in haploidentical hematopoietic stem-cell transplantation. Blood. 2007 Oct 1;110(7):2764-7. doi: 10.1182/blood-2007-04-087056. Epub 2007 Jul 16.
Background: PubMed 18673358Fang B, Li N, Song Y, Li J, Zhao RC, Ma Y. Cotransplantation of haploidentical mesenchymal stem cells to enhance engraftment of hematopoietic stem cells and to reduce the risk of graft failure in two children with severe aplastic anemia. Pediatr Transplant. 2009 Jun;13(4):499-502. doi: 10.1111/j.1399-3046.2008.01002.x. Epub 2008 Jul 30.
Background: PubMed 20380540Wang H, Wang Z, Xue M, Liu J, Yan H, Guo Z. Co-transfusion of haplo-identical hematopoietic and mesenchymal stromal cells to treat a patient with severe aplastic. Cytotherapy. 2010 Jul;12(4):563-5. doi: 10.3109/14653241003695059.

Related trials for Severe Aplastic Anemia

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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.

Yang Xiao, MD study chair

Guangzhou General Hospital of Guangzhou Military Command

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 in people with Severe Aplastic Anemia, run by Guangzhou General Hospital of Guangzhou Military Command 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 10 inclusion and 8 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 Sep 2014. 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

survival rate (time frame: up to 2 years after HSCT). 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 NCT02247973, which also lists 15 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 25 Sep 2014.