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Acute Graft-versus-host DiseasePhase 2NCT01765634

Mesenchymal Stem Cells for Treatment of Refractory Acute Graft-versus-host Disease

A Phase 2 study testing Mesenchymal stem cells and Non-mesenchymal stem cells in people with Acute Graft-versus-host Disease, run by Nanfang Hospital, Southern Medical University 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 The sponsor has not verified this record for over two years, so it may be out of date.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
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Trial summary

The purpose of this study is to evaluate the utility of treating patients experiencing refractory acute graft-versus-host disease with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. Read more about this study

Who is it for?
Ages 12 to 65 years
Conditions the study is for
Acute Graft-versus-host Disease
What is being tested?
Mesenchymal stem cells and Non-mesenchymal stem cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
1 siteChina
Study dates
Jan 2013to Dec 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 12 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • A patient age of 12-65 years
  • Recipients of allogeneic hematopoietic stem cell transplantation
  • Patients with refractory aGVHD
  • On a voluntary basis, patients are divided into MSCs and Non-MSCs group
  • 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

  • Department of Hematology,Nanfang Hospital, Southern Medical University

    Guangzhou, Guangdong, 510515

    Li Xuan, +86-020-61641613, 356135708@qq.com

    Qifa Liu

    Recruiting

Other trials in China

About this study

Official title: Mesenchymal Stem Cells From Third-party Donors for Treatment of Refractory Acute Graft-versus-host Disease

The purpose of this study is to evaluate the utility of treating patients experiencing refractory acute graft-versus-host disease with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. The objective was to evaluate the effect and safety of such treatment on refractory acute graft-versus-host disease.

Read the detailed description

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) can cure many hematologic diseases. Although great progress has been made in the prevention and treatment of side effects associated with transplantation,acute graft-versus-host disease(aGVHD) remains an important complication that occurs in 35-80% patients. The mortality of aGVHD is positively correlated with its severity. At present, glucocorticoids is still the first line treatment of aGVHD. If glucocorticoids treatment is ineffective, second line drugs would be taken, such as tacrolimus(FK506), mycophenolate mofetil (MMF)and antithymocyte globulin (ATG). However, no method could be continuously effective. The effective rates of second line treatment to aGVHD is only about 60%. The effective rates and prognosis of refractory aGVHD are even worse.

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 GVHD after allo-HSCT and so on. However, the efficacy of treatment of refractory aGVHD using expanded BM-derived MSCs from a third-party donor is rarely reported. 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 in treating patients with refractory aGVHD.

Being tested
Mesenchymal stem cells, Non-mesenchymal stem cells
Keywords
Hematopoietic Stem Cell Transplantation, Mesenchymal Stem Cells, Acute Graft-versus-host disease

What participants receive

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

  • Mesenchymal stem cellsBiological

    Also called Mscs

    Mesenchymal stem cells will be intravenously infused via a central venous catheter(at a dose of 1×10^6 cells/kg, over 15 min) each week, four times for a cycle.

  • Non-mesenchymal stem cellsBiological

    Also called Non-MSCs

    Other second line drugs are taken.

Study groups in NCT01765634
Study groupRoleWhat they receive
Mesenchymal stem cellsExperimentalMesenchymal stem cell group refers to treatment with mesenchymal stem cells (1×10^6 cells/kg, intravenously)each week, four times for a cycle
Receives: Biological: Mesenchymal stem cells
Non-mesenchymal stem cellsExperimentalNon-mesenchymal stem cell group refers to treatment with other second line drugs
Receives: Biological: Non-mesenchymal stem cells

What is being measured

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

  1. The efficacy of treatment for refractory aGVHD

    The response criteria include complete response (CR), part response (PR), stable disease(SD) and progressive disease(PD). CR:aGVHD symptoms and signs disappear; PR:aGVHD symptoms and signs improve; SD:aGVHD symptoms and signs remain (without improvement or deterioration);PD: aGVHD symptoms and signs deteriorate.

    Measured: 1 year

Secondary outcomes (1)

  1. acute and late toxic side effects of 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.

  1. First posted10 Jan 2013 (estimated)
  2. Study startJan 2013
  3. Primary completionDec 2015 (estimated)
  4. Study completionDec 2016 (estimated)

Resources

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01765634
Sponsor's protocol ID
NFH-MSCs-aGVHD-2013
Status last verified
Jan 2013
First submitted
5 Jan 2013
Last updated
10 Jan 2013
Results posted
No
Data monitoring committee
Yes

Publications

  • 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

  • von Bonin M, Stolzel F, Goedecke A, Richter K, Wuschek N, Holig K, Platzbecker U, Illmer T, Schaich M, Schetelig J, Kiani A, Ordemann R, Ehninger G, Schmitz M, Bornhauser M. Treatment of refractory acute GVHD with third-party MSC expanded in platelet lysate-containing medium. Bone Marrow Transplant. 2009 Feb;43(3):245-51. doi: 10.1038/bmt.2008.316. Epub 2008 Sep 29.

    PubMed 18820709

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Mesenchymal stem cells and Non-mesenchymal stem cells in people with Acute Graft-versus-host Disease, 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 5 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 10 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?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The efficacy of treatment for refractory aGVHD (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 NCT01765634, 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 10 Jan 2013.

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