Umbilical Cord Derived Mesenchymal Stem Cells Transplantation for Active and Refractory Systemic Lupus Erythematosus

Study Overview
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Brief Summary
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This study will explore safety and efficacy of allogeneic umbilical cord (UC) derived mesenchymal stem cells transplantation (MSCT) to treat patients with active and refractory systemic lupus erythematosus (SLE) who have been resistant to multiple standard treatments. The underlying hypothesis is that the active SLE condition is caused by an abnormal immune homeostasis that can be restored by MSCT.

Detailed Description
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Inclusion Criteria: All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 70 years old, SLEDAI≥8; Lupus nephritis with 24h urine protein≥1g; Refractory disease as determined by failure of the following regimens: Trial of corticosteroids (oral prednisone more than 20 mg/day); Trial of cyclophosphamide 0.4   0.6 / m2 every two weeks for six months, or other immunosuppressive drugs, such as mycophenolate mofetil 2 g / day, for three months; Patients must sign an informed consent indicating that they are aware of the investigational nature of the study in keeping with the policy of the hospital; Willing to use contraception throughout the study and for 12 months following treatment

Official Title
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Phase I/II: Umbilical Cord Derived Mesenchymal Stem Cells Transplantation For Active And Refractory Systemic Lupus Erythematosus

Conditions
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Systemic Lupus Erythematosus

Intervention / Treatment
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* Biological: human umbilical cord derived MSC transplantation for SLE


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All patients fulfilled the American College of Rheumatology (ACR) criteria of SLE, man or woman aged from 15 to 60 years old, SLEDAI≥8; Lupus nephritis with 24h urine protein≥1g; Refractory disease as determined by failure of the following regimens: Trial of corticosteroids (oral prednisone more than 20 mg/day); Trial of cyclophosphamide 0.4   0.6 / m2 every two weeks for six months, or other immunosuppressive drugs, such as mycophenolate mofetil 2 g / day, for three months; Patients must sign an informed consent indicating that they are aware of the investigational nature of the study in keeping with the policy of the hospital; Willing to use contraception throughout the study and for 12 months following treatment Exclusion Criteria: Abnormal liver function (ALT higher than 3 times the normal value); End-stage renal failure; Severe heart and pulmonary failure, or other important organs damage; Uncontrolled infections Pregnant or breast feeding women, male or female who intended to recent pregnancy

Ages Eligible for Study
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Minimum Age: 15 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: human umbilical cord derived MSC<br>human umbilical cord derived MSC transplantation for SLE | Biological: human umbilical cord derived MSC transplantation for SLE<br> <br> * Other names: Allogenic MSC derived from umbilical cord;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| British Isles Lupus Assessment Group score (BILAG) |  | up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Lupus serology (Alb, ANA, dsDNA, C3, C4) |  | pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation | 
| Renal function (GFR, Blood Urea Nitrogen, urinalysis) |  | pre-MSC transplantation, 1, 3, 6 and 12 months post MSC transplantation (for GFR assessed at baseline and 12 months after MSCT) |