Allogeneic Mesenchymal Stem Cells Transplantation for Systemic Sclerosis (SSc)

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

Detailed Description
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To test a new approach using allogeneic derived mesenchymal stem cell based therapy (MSCT) to treat refractory SSc To determine the disease-free survival in SSc patients treated with MSCT To assess adverse events of allogeneic MSC transplantation To assess the association of remission for organ function, clinical score and SSc serology levels at baseline with disease-free survival

Conditions
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Systemic Sclerosis, Mesenchymal Stem Cells

Intervention / Treatment
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* Biological: Allogeneic Mesenchymal Stem Cells (AlloMSC)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All patients fulfilled the American College of Rheumatology (former American Rheumatism Association - ARA) for SSc Rapidly progressive disease <2 years duration with a modified Rodnan skin score(mRSS) above 20, plus ESR >25 mm/first h and/or Hb <11 g/dL, not explained by other causes than active SSc lung involvement: with a vital capacity (VC) or DLCO below 70% predicted, or a mean pulmonary artery pressure (PAP) above 40 mmHg (measured by echocardiography) digestive tract involvement: with serum albumin ,25 g/L or weight loss exceeding 10% body weight in the preceding year kidney involvement: with 24-h urinary protein above 0.5 g or serum creatinine above 120 mmol/L Exclusion Criteria: Uncontrolled arrhythmia, echocardiographic left ventricular ejection fraction (LVEF) <50% or mean PAP >50 mmHg, DLCO<45% of predicted Creatinine clearance <20 ml/min Platelets<80 000/mm3, haemorrhagic cystitis (4) HIV or HTLV1 seropositivity, malignancy, pregnancy, a cardiac or vascular prosthesis, and no vascular access

Ages Eligible for Study
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Minimum Age: 15 Years
Maximum Age: 65 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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: Allogeneic Mesenchymal Stem Cells (AlloMSC)|Allogeneic mesenchymal stem cells will be infused intravenously (single dose, 10^6 cells/kg body weight).|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| mRSS score,HRQOL score, SF-36 score for SSc patients |  | monthly | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Remission for organ function, VC, DLCO, PAP, serum albumin, serum creatitin, weight loss, 24h proteinuria |  | every three month | 
| SSc Serology(ATA,ACA,ANA,anti-ssDNA,anti-dsDNA,IgM,IgG,and IgA,complement C3 and C4 |  | every three month | 
| Change of peripheral blood B and T cells |  | every three month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Systemic sclerosis, Mesenchymal Stem Cells, Transplantation