Allogeneic Mesenchymal Stem Cells Transplantation for Systemic Sclerosis (SSc)

Sponsor

The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School (Other)

Overall Status

Unknown status

CT.gov ID

NCT00962923

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Systemic Sclerosis Mesenchymal Stem Cells	Biological: Allogeneic Mesenchymal Stem Cells (AlloMSC)	Phase 1/Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Study Start Date :

Aug 1, 2009

Anticipated Primary Completion Date :

Dec 1, 2010

Anticipated Study Completion Date :

Dec 1, 2011

Outcome Measures

Primary Outcome Measures

mRSS score,HRQOL score, SF-36 score for SSc patients [monthly]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

15 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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