Allogeneic Mesenchymal Stem Cells Transplantation for Systemic Sclerosis (SSc)

Phase 1

• Conditions: Systemic Sclerosis; Mesenchymal Stem Cells

• Registration Number: NCT00962923
• Lead Sponsor: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School

Brief Summary

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

* 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

Study Timeline

• Status Verified: 2009-08
• Study Start: 2009-08
• Study First Submitted: 2009-08-19
• Study First Submitted QC: 2009-08-19
• Last Update Submitted: 2009-08-19
• Study First Posted: 2009-08-20
• Last Update Posted: 2009-08-20
• Primary Completion: 2010-12
• Study Completion: 2011-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
mRSS score,HRQOL score, SF-36 score for SSc patients	monthly

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

the Affiliated Drum Tower Hospital of Nanjing University Medical School; 🇨🇳 Nanjing, Jiangsu, China