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Acute GVH DiseasePhase 1/2NCT01754454

Safety and Efficacy of UC-MSC in Patients With Acute Severe Graft-versus-host Disease

A Phase 1/2 study testing Human Umbilical Cord Derived MSC in people with Acute GVH Disease, run by Affiliated Hospital to Academy of Military Medical Sciences at 2 sites 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 The sponsor has not verified this record for over two years, so it may be out of date.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

Allogenic haemopoietic stem cell transplantation (allo-HSCT) is the treatment for many malignant and non-malignant hematologic disorders. Acute graft-versus-host disease (aGVHD) is a serious life-threatening complication after allo-HSCT. The outcome for patients with aGVHD is poor and overall survival is low. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Acute GVH Disease
What is being tested?
Human Umbilical Cord Derived MSC
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
2 sitesChina
Study dates
Dec 2012to Dec 2016
Phase
Phase 1/2
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • aged 18-70 years
  • undergoing allogeneic hematopoietic stem cell transplant recipients
  • post-transplant acute GVHD (Ⅲ ~ Ⅳ degrees)
  • the effects of other immunosuppressive therapy
  • glucocorticoid resistance or glucocorticoid therapy invalid
  • cooperation observed adverse events and efficacy
  • patients understand the status of the experimental observations, with the doctor's treatment and post-treatment follow-up, patients or their legal representatives signed a written informed consent

Who cannot join

  • had severe allergy history
  • within three months to participate in other medical or drug trials
  • as a subjects was sampled within three months
  • smoking, alcohol and drug abusers
  • suffers from an important organ of primary disease (heart, liver, kidney, lung, brain, etc.), infectious diseases (including HIV and suspicious latent infection), people with disabilities and / or mental disorders
  • to judge according to the researchers, the subjects could not complete the study or may not be able to comply with the requirements of this study (due to administrative reasons or other reasons)
Show all 20Show fewer
  • Exit criteria:
  • In clinical trials, the participants had some complications, complications or special physiological changes, unfit to continue to accept the test
  • subjects with poor compliance, affect the determination of the pharmacokinetic results
  • subjects using the drugs may affect the results of the pharmacokinetic
  • the occurrence of serious adverse events in subjects
  • subjects withdrew informed consent, or withdraw from the trial itself
  • Termination criteria:
  • serious adverse events (except the researchers judgment and research drugs must be independent), should stop all test
  • found that clinical research plan has important lapses, difficult to evaluate the effects of drugs, or a better design in implementation had an important deviation, difficult to continue to evaluate the effects of drugs
  • sponsor for the request to terminate (such as funds reason, management reason, etc.)
  • state food and drug administration for some reason ordered to terminate test
  • does not meet the entry criteria
  • once the drug is not used in
  • no records

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • Department of Hematopoietic Stem Cell Transplantation

    Beijing, 100071

  • Department of Hematopoietic Stem Cell Transplantation, Affiliated Hospital to Academy of Military Medical Sciences

    Beijing

Other trials in China

About this study

Official title: Open-Label, Single-Center, Self Control, Phase Ⅰ/Ⅱ Clinical Trial to Evaluate the Safety and the Efficacy of Umbilical Cord-derived Mesenchymal Stem Cells in Patients With Acute Graft-versus-host Disease

Allogenic haemopoietic stem cell transplantation (allo-HSCT) is the treatment for many malignant and non-malignant hematologic disorders. Acute graft-versus-host disease (aGVHD) is a serious life-threatening complication after allo-HSCT. The outcome for patients with aGVHD is poor and overall survival is low. Acute graft-versus-host disease (GVHD), as the major complication of allogeneic peripheral blood stem cell transplantation(PBSCT), limits the application of this curative therapy. Mesenchymal stem cells (MSCs) are multipotent stem cells, which are able to modulate immune response in vitro and in vivo, and have possibilities of treating diseases caused by immune dysregulation such as aGVHD. MSCs obtained from umbilical cord (UC) have similar immunosuppressive properties as bone marrow-MSCs. In addition, UC-derived MSCs can be used for off-the-shelf use and are obtained without any harm to donors than bone marrow-MSCs. Therefore, the investigators designed this study to evaluate the safety and efficacy of UC-derived MSCs in patients with aGVHD.

Read the detailed description

The mesenchymal stem cells (MSC) is a class of low immunogenicity from mesoderm, had the self-renewal and differentiation potential as pluripotent stem cells. The umbilical cord is one of the important sources of MSC. Human UC-MSC compared with the bone marrow-derived MSC has the following advantages: (1) the umbilical cord, as a "waste" after giving birth can be obtained without any harm to the donor. (2) easy available, and no ethical issues. (3) its likelihood of contamination is small because of the protection of the placental barrier. (4) human UC-MSC has a stronger amplification capacity, a shorter doubling time, a higher colony forming efficiency and lower immunogenicity. (5) which avoids the activity and differentiation of bone marrow-derived MSC latent with the donor disadvantage of increasing age decreased.

human UC-MSC not only for hematopoietic stem cell graft implantation and amplification has a role in promoting and exhibit immunomodulatory effects and characteristics of damaged or targeted sites of chronic inflammation. human UC-MSC in the prevention and treatment of graft-versus-host disease (GVHD) induced organ transplantation immune tolerance and other fields has broad application prospects.

After more than 10 years of exploration, the investigators and other practitioners caught studies found that the umbilical cord is more suitable as mesenchymal stem cells seed cells relative to the bone marrow, umbilical cord easily obtained, no ethical issues, better amplification ability in vitro, viral contamination risk is small, and many other advantages. Therefore, over the years, the investigator team for the umbilical cord mesenchymal stem cells in a lot of the basic work has been confirmed that mesenchymal stem cells derived from the umbilical cord had safety and effectiveness in in the treatment of acute graft versus host disease in zoology test, and successfully establish a perfect umbilical cord mesenchymal stem cells in standard operating procedures (including screening umbilical cord, large-scale preparation, quality inspection, preservation, recovery, transportation, infusion, etc.). The investigators will carry out the clinical research based on above preliminary results.

Being tested
Human Umbilical Cord Derived MSC

What participants receive

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

  • Human Umbilical Cord Derived MSCBiological

    Also called UC-MSC

    1×10^6 UC-MSCs per kg intravenous injection the number of infusions: once a week, for four weeks in a row Time interval: estimate the condition of follow-up visit afer 28 days

Study groups in NCT01754454
Study groupRoleWhat they receive
Human Umbilical Cord Derived MSCExperimentalHuman Umbilical Cord Derived MSC: Patients who receive standard of care plus treatment with ex vivo cultured adult human Umbilical Cord Derived Mesenchymal Stem Cells
Receives: Biological: Human Umbilical Cord Derived MSC

What is being measured

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

  1. Safety of UC-MSC in patients with acute graft-versus-host disease

    • vital signs
    • adverse events related to infusion
    • physical examination indexes
    • Eastern Cooperative Oncology Group(ECOG)performance status
    • mixed lymphocyte reaction(MLR)

    Measured: 180 days

Secondary outcomes (1)

  1. Efficacy of UC-MSC in patients with acute graft-versus-host disease

    • Complete, partial response rate at 28 and 180 days
    • Complete response(CR)rate (%)=(number of CR/number of participants)*100%
    • Partial response(PR)rate (%)=(number of PR/number of participants)*100%

    Measured: 180 days

Timeline

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

  1. First posted21 Dec 2012 (estimated)
  2. Study startDec 2012
  3. Primary completionDec 2016 (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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01754454
Sponsor's protocol ID
307-CTC-MSC-002
Status last verified
Feb 2016
First submitted
11 Nov 2012
Last updated
8 Apr 2016
Results posted
No
Data monitoring committee
Yes

Publications

  • Niu JW, Li Y, Xu C, Sheng H, Tian C, Ning H, Hu J, Chen J, Li B, Wang J, Lou X, Liu N, Su Y, Sun Y, Qiao Z, Wang L, Zhang Y, Lan S, Xie J, Ren J, Peng B, Wang S, Shi Y, Zhao L, Zhang Y, Chen H, Zhang B, Hu L. Human umbilical cord-derived mesenchymal stromal cells for the treatment of steroid refractory grades III-IV acute graft-versus-host disease with long-term follow-up. Front Immunol. 2024 Aug 15;15:1436653. doi: 10.3389/fimmu.2024.1436653. eCollection 2024.

    PubMed 39211037

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Human Umbilical Cord Derived MSC in people with Acute GVH Disease, run by Affiliated Hospital to Academy of Military Medical Sciences at 2 sites 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 7 inclusion and 20 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 8 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Safety of UC-MSC in patients with acute graft-versus-host disease (time frame: 180 days). 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 NCT01754454, which also lists 1 linked publication.

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 8 Apr 2016.

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