Clinical trial NCT00953485: Allogeneic Mesenchymal Stem Cells Transplantation for Primary Sjögren's Syndrome (pSS)
Official title: Clinical Trial of Allogeneic Mesenchymal Stem Cells Transplantation for Primary Sjögren's Syndrome - Phase Ⅰ/Ⅱ
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Sjogren's Syndrome
Mesenchymal Stem Cells
Keywords: Sjogren's Syndrome
Mesenchymal Stem Cells
Transplantation
Lead sponsor: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Sponsor class: OTHER
First posted: 2009-08-06
Enrollment: 20
Sex: ALL
Minimum age (years): 15
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will explore safety and efficacy of allogeneic mesenchymal stem cells transplantation (MSCT) to treat patients with diagnosis of primary Sjögren's Syndrome (pSS) who have been resistant to multiple standard treatments. The underlying hypothesis is that the pSS 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 primary Sjögren's Syndrome (pSS)
* To determine the disease-free survival in pSS patients treated with MSCT in terms of disease activity index, pSS serology and salivary gland function
* To assess adverse events of allogeneic MSC transplantation
* To assess the association of disease activity index, pSS serology levels, and saliva flow rate at baseline with disease-free survival

Eligibility criteria: Inclusion Criteria:

* All patients fulfilled the international classification criteria (2002) for primary Sjögren's Syndrome, man or woman aged from 15 to 70 years old, Sjögren's Syndrome Disease Activity Index (SSDAI)≥8.
* Stimulated whole saliva flow rate less than 1\~6ml/6min.
* Patients must be informed of the investigational nature of this study and give written informed consent in accordance with the institutional and hospital guidelines.
* Women of reproductive potential may not participate unless they have agreed to use an effective contraceptive method while on this trial and for 12 months following treatment. Women of child-bearing potential must have a pregnancy test performed within 72 hours prior to initiation of treatment.

Exclusion Criteria:

* End-stage renal failure.
* Severe cardiopulmonary compromise, or other system failure.
* Active, uncontrolled infections.
* Pregnant or nursing women may not participate due to the possibility of fetal harm or harm to nursing infants from this treatment regimen.

Primary outcomes:
measure: Sjögren's syndrome disease activity index
timeFrame: Monthly
Secondary outcomes:
measure: pSS Serology (ANA, dsDNA, SS-A, SS-B)
timeFrame: Monthly
measure: Improvement of salivary gland function (measured as stimulated saliva flow rate)
timeFrame: Monthly
Other outcomes:
Locations:
city: Nanjing
country: China
facility: the Affiliated Drum Tower Hospital of Nanjing University Medical School
state: Jiangsu
Study officials:
affiliation: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
name: Lingyun Sun, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sun L, Akiyama K, Zhang H, Yamaza T, Hou Y, Zhao S, Xu T, Le A, Shi S. Mesenchymal stem cell transplantation reverses multiorgan dysfunction in systemic lupus erythematosus mice and humans. Stem Cells. 2009 Jun;27(6):1421-32. doi: 10.1002/stem.68.
citation: Liang J, Zhang H, Hua B, Wang H, Wang J, Han Z, Sun L. Allogeneic mesenchymal stem cells transplantation in treatment of multiple sclerosis. Mult Scler. 2009 May;15(5):644-6. doi: 10.1177/1352458509104590.
citation: Zhou K, Zhang H, Jin O, Feng X, Yao G, Hou Y, Sun L. Transplantation of human bone marrow mesenchymal stem cell ameliorates the autoimmune pathogenesis in MRL/lpr mice. Cell Mol Immunol. 2008 Dec;5(6):417-24. doi: 10.1038/cmi.2008.52.
citation: Sun LY, Zhang HY, Feng XB, Hou YY, Lu LW, Fan LM. Abnormality of bone marrow-derived mesenchymal stem cells in patients with systemic lupus erythematosus. Lupus. 2007;16(2):121-8. doi: 10.1177/0961203306075793.
citation: Xu J, Wang D, Liu D, Fan Z, Zhang H, Liu O, Ding G, Gao R, Zhang C, Ding Y, Bromberg JS, Chen W, Sun L, Wang S. Allogeneic mesenchymal stem cell treatment alleviates experimental and clinical Sjogren syndrome. Blood. 2012 Oct 11;120(15):3142-51. doi: 10.1182/blood-2011-11-391144. Epub 2012 Aug 27.