NCT02291770

Brief Summary

Chronic Graft-versus-Host Disease (cGvHD) is a potentially lethal disorder. A variety of second line immunosuppressive agents have been investigated but no optimal treatment has emerged. There is therefore a need for novel treatment strategies. Mesenchymal stromal cells (MSC) exhibit immunomodulatory properties and a recent pilot study suggests a response rate of 70% in steroid- refractory patients. In the present randomized study the efficacy and safety of MSC treatment will be further studied in patients with cGvHD.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
130

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Oct 2014

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2014

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

November 5, 2014

Completed
9 days until next milestone

First Posted

Study publicly available on registry

November 14, 2014

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2015

Completed
4.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2019

Completed
Last Updated

November 14, 2014

Status Verified

November 1, 2014

Enrollment Period

1.1 years

First QC Date

November 5, 2014

Last Update Submit

November 11, 2014

Conditions

Chronic Graft-Versus-Host Disease

Keywords

Chronic Graft-Versus-Host DiseaseMesenchymal Stromal Cells

Outcome Measures

Primary Outcomes (1)

  • Proportion of patients responding to treatment of cGvHD with MSC

    90 days

Secondary Outcomes (5)

  • Overall survival

    2 year

  • Progression-free survival

    2 year

  • Time without systemic immunosuppression

    2 year

  • Cumulative incidents of non-relapse mortality

    2 year

  • Adverse events

    2 year

Other Outcomes (2)

  • Quality of life

    2 year

  • Immune reconstitution including monitoring of absolute T-cell subsets, B-cells, NK-cells as well as biomarkers of cGvHD

    2 year

Study Arms (2)

Mesenchymal stem cells (MSC)

ACTIVE COMPARATOR

Patients with newly diagnosed cGvHD receive primary treatment plus MSC: 1. MSC+prednisone+cyclosporine; 2. MSC+prednisone+tacrolimus; 3. MSC+prednisone+mycophenolate mofetil.

Biological: Mesenchymal Stromal Cells

Placebo

PLACEBO COMPARATOR

Patients with newly diagnosed cGvHD receive primary treatment: 1. Placebo+prednisone+cyclosporine; 2. Placebo+prednisone+tacrolimus; 3. Placebo+prednisone+mycophenolate mofetil.

Interventions

Mesenchymal Stromal CellsBIOLOGICAL

Mesenchymal stem cell(MSC). Patients with newly diagnosed cGvHD: prednisone 1mg/kg + cyclosporine or tacrolimus and MSC 2Ă—1,000,000 MSC/kg, IV twice a week for the first two weeks and weekly for the following two weeks(6 doses totally).

Also known as: MSC
Mesenchymal stem cells (MSC)

Eligibility Criteria

Age14 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Newly diagnosed cGvHD
  • Informed consent obtained from patient and donor.
  • Any patient who has undergone allogeneic stem cell transplantation with c GvHD.
  • Have not received additional agent for cGVHD within 3 months.
  • Expected life is more than 90 days.
  • Adequate pulmonary function with no evidence of chronic obstructive or severe restrictive pulmonary disease.
  • Adequate cardiac function with no evidence of uncontrolled high blood pressure,congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to the process.

You may not qualify if:

  • Invasive fungal disease.
  • Active cytomegalovirus (CMV)/Epstein-Barr virus(EBV)/varicella disease).
  • Patient is with a history of hypersensitivity to bovine products.
  • Relapsed malignancy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Xin Du

Guangzhou, Guangdong, 510080, China

RECRUITING

Related Publications (2)

  • Weng J, He C, Lai P, Luo C, Guo R, Wu S, Geng S, Xiangpeng A, Liu X, Du X. Mesenchymal stromal cells treatment attenuates dry eye in patients with chronic graft-versus-host disease. Mol Ther. 2012 Dec;20(12):2347-54. doi: 10.1038/mt.2012.208. Epub 2012 Oct 16.

    PMID: 23070118BACKGROUND

  • Weng JY, Du X, Geng SX, Peng YW, Wang Z, Lu ZS, Wu SJ, Luo CW, Guo R, Ling W, Deng CX, Liao PJ, Xiang AP. Mesenchymal stem cell as salvage treatment for refractory chronic GVHD. Bone Marrow Transplant. 2010 Dec;45(12):1732-40. doi: 10.1038/bmt.2010.195. Epub 2010 Sep 6.

    PMID: 20818445BACKGROUND

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Organizing PneumoniaBronchiolitis ObliteransBronchiolitisBronchitisBronchial DiseasesRespiratory Tract DiseasesLung Diseases, ObstructiveLung DiseasesGraft vs Host DiseaseImmune System Diseases

Study Officials

  • Xin Du, Prof.

    Guangdong Provincial People's Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Xin Du, Prof.

CONTACT

+86 02083827812-62122miyadu@hotmail.com

Jianyu Weng, Prof.

CONTACT

+86 02083827812-62122wengjianyu1969@163.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 5, 2014

First Posted

November 14, 2014

Study Start

October 1, 2014

Primary Completion

November 1, 2015

Study Completion

December 1, 2019

Last Updated

November 14, 2014

Record last verified: 2014-11

Locations

CN(1)