Safety and Efficacy Study of Allogenic Mesenchymal Stem Cells to Treat Extensive Chronic Graft Versus Host Disease

NCT00972660 | Unknown Phase 2 DMC

• 1 other identifier: GDREC.[2009]008
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

Study Design: Treatment, Randomized, Open Label, Parallel Assignment,Safety/Efficacy Study. The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSC) expanded ex-vivo infusion for the treatment of patients who have developed a newly diagnosed extensive or refractory chronic graft versus host disease (chronic GVHD) to the usual therapeutic measures.

Conditions

Graft Versus Host Disease

Keywords

Mesenchymal stem cell; Efficacy; Safety

Outcome Measures

Primary Outcomes (1)

• The total Response rate defined as patients with complete and partial response.

  Within the first 3 months (plus or minus 7 days) after randomization

Secondary Outcomes (4)

• Overall Survival

  Randomization until death or two years post last subject last treatment visit (or clinical cutoff)

• Events Free Survival

  Randomization until death or two years post last subject last treatment visit (or clinical cutoff)

• The percentage of patients who can taper or discontinue the immunosuppressive agents

  Randomization untill two years post the last subject last treatment visit (or clinical cutoff)

• Serum cytokine levels and lymphocyte subsets in patients with chronic GVHD

  Achieve best response within the first 3 months after randomization

Study Arms (2)

Control group

ACTIVE COMPARATOR

Patients with newly diagnosed extensive cGVHD receive prednisone and cyclosporine or tacrolimus. Patients with refractory extensive cGVHD receive primary treatment (eg,prednisone and cyclosporine or tacrolimus, or plus mycophenolate mofetil, or methotrexate.)

Drug: Prednisone and cyclosporine or primary therapies

Mesenchymal stem cell (MSC)

EXPERIMENTAL

Patients with newly diagnosed extensive cGVHD receive MSC plus prednisone and cyclosporine or tacrolimus. Patients with refractory extensive cGVHD receive MSC plus their primary immunosuppressive treatment (eg. prednisone + cyclosporine or tacrolimus, or plus mycophenolate mofetil, or plus methotrexate.)

Biological: Mesenchymal stem cell (MSC)

Interventions

Mesenchymal stem cell (MSC) BIOLOGICAL

Experimental:Mesenchymal stem cell(MSC). Patients with newly diagnosed extensive cGVHD: prednisone 1mg/kg + cyclosporine or tacrolimus and MSC 2×1,000,000 MSC/kg, IV twice a week for the first two weeks and weekly for the following two weeks(6 doses totally). Refractory extensive cGVHD: receive primary treatment (prednisone + cyclosporine or tacrolimus, or plus mycophenolate mofetil, or plus methotrexate ) and MSC2×1,000,000 MSC/kg, IV twice a week for the first two weeks and weekly for the following two weeks(6 doses totally).

Also known as: Medrol, Sandimmun Neoral, FK506,prograf, Cellcept, MTX

Mesenchymal stem cell (MSC)

Prednisone and cyclosporine or primary therapies DRUG

Patients with newly diagnosed extensive cGVHD: prednisone 1mg/kg + cyclosporine or tacrolimus Patients with refractory extensive cGVHD: primary treatment (eg.prednisone 1mg/kg + cyclosporine or tacrolimus,or plus mycophenolate mofetil, or methotrexate.)

Also known as: Medral, Sandimmun Neoral, FK506,Prograf, Cellcept,MMF, MTX

Control group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed consent obtained from patient and donor.
• Any patient who has undergone allogeneic stem cell transplantation with extensive chronic 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.

Sponsors & Collaborators

• Guangdong Provincial People's Hospital: lead
• Sun Yat-sen University: collaborator

Study Sites (1)

Guangdong General Hospital

Guangzhou, Guangdong, 510080, China

Location

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

Methylprednisolone; Cyclosporine; Tacrolimus; Mycophenolic Acid; Prednisone

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Macrolides; Lactones; Organic Chemicals; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Pregnadienediols

Study Officials

• Xin Du, MD.PhD.

  Guangdong Provincial People's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 4, 2009
• First Posted: September 7, 2009
• Study Start: September 1, 2009
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2017
• Last Updated: August 26, 2014

Record last verified: 2010-05

Locations

CN (1)