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

NCT01754454 | Unknown Phase 1 DMC

• 1 other identifier: 307-CTC-MSC-002
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief 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. 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.

Conditions

Acute GVH Disease

Outcome Measures

Primary Outcomes (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)

  180 days

Secondary Outcomes (1)

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

  180 days

Study Arms (1)

Human Umbilical Cord Derived MSC

EXPERIMENTAL

Human Umbilical Cord Derived MSC: Patients who receive standard of care plus treatment with ex vivo cultured adult human Umbilical Cord Derived Mesenchymal Stem Cells

Biological: Human Umbilical Cord Derived MSC

Interventions

Human Umbilical Cord Derived MSC BIOLOGICAL

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

Also known as: UC-MSC

Human Umbilical Cord Derived MSC

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Affiliated Hospital to Academy of Military Medical Sciences: lead

Study Sites (2)

Department of Hematopoietic Stem Cell Transplantation

Beijing, 100071, China

Location

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

Beijing, China

Location

Related Publications (1)

• 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.

  PMID: 39211037 DERIVED

Study Officials

• Hu Chen, M.D., Ph.D.

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

  STUDY CHAIR

• Bin Zhang, M.D. Ph.D

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

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2012
• First Posted: December 21, 2012
• Study Start: December 1, 2012
• Primary Completion: December 1, 2016
• Study Completion: December 1, 2016
• Last Updated: April 8, 2016

Record last verified: 2016-02

Locations

CN (2)