Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Ankylosing Spondylitis (AS)
The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for the therapy of Ankylosing spondylitis (AS)
Biological: Human umbilical cord-derived MSCs
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat AS|
- The Assessment of Spondyloarthritis International Society (ASAS)20 response [ Time Frame: 1 year ] [ Designated as safety issue: No ]
ASAS measures symptomatic improvement in AS patients.ASAS=4 domains:patient global assessment of disease activity,pain,function,inflammation.ASAS 20=20% improvement(vs.baseline)and an abosolute change≥1 units on a 0-10 scale(0=no disease activity;10=high disease activity)for ≥3 domains,and no worsening in remaining domain.
Patient global Pain Function (as measured by the Bath Ankylosing Spondylitis Functional Index - BASFI) Inflammation (mean of the Bath Ankylosing Spondylitis Disease Activity Index - BASDAI question 5 and 6)
- erythrocyte sedimentation rate (ESR) [ Time Frame: 1 year ] [ Designated as safety issue: No ]erythrocyte sedimentation rate (ESR) level will be mainly observed after transplanting 3, 6,12-month.
- imageology [ Time Frame: 1 year ] [ Designated as safety issue: No ]imageology will be mainly observed after transplanting 3, 6,12-month.
- C-reactive protein (CRP) [ Time Frame: 1 year ] [ Designated as safety issue: No ]C-reactive protein (CRP) level will be mainly observed after transplanting 3, 6,12-month.
- Percentage of systemic T regulatory cell population [ Time Frame: 1 year ] [ Designated as safety issue: No ]Percentages of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year
- Side effects [ Time Frame: 1 year ] [ Designated as safety issue: No ]Side effects were observed after the treatment
|Study Start Date:||January 2011|
|Estimated Study Completion Date:||December 2013|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Experimental: Human umbilical cord-derived MSCs and DMARDs
Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated after three months and DMARDs such as sulfasalazine,methotrexate,thalidomide po for 12 months
Biological: Human umbilical cord-derived MSCs
1.0E+6 MSC/kg, IV drop and repeat repeated after three months
No Intervention: DMARDs
DMARDs such as sulfasalazine,methotrexate,thalidomide po for 12 months
Ankylosing spondylitis (AS) is a chronic, progressive inflammatory rheumatic disease involving primarily the sacroiliac joints and the axial skeleton. The main clinical features are back pain and progressive stiffness of the spine. Oligoarthritis of the hips and shoulders, enthesopathy, and anterior uveitis are common, and involvement of the heart and lungs is rare. The current understanding of the pathogenesis of this disorder is limited.It mainly about to hereditary susceptibility (eg hla-b27),infection and autoimmunity.
Although traditional drugs, such as Nonsteroidal antiinflammatory drugs (NSAIDs) disease-modifying antirheumatic drugs (DMARDs such as MTX,SASP OR thalidomide) and steroids have been used in the treatment of AS, however, many studies have indicated that the overall response to these drugs is not satisfied. Addition, the severe side effects of these drugs have also been observed. The management of AS patients therefore remains unsatisfactory and targeted therapies are needed. Human MSCs isolated from human umbilical cord/placenta have been shown to have immunoregulatory, immunosuppressive, stimulating hematopoiesis and tissue repairing properties. This study will evaluate the safety and effectiveness of MSC transplantation in the AS patients.
This study will last 2 to 3 years. Participants will be randomly assigned to receive either MSC transplant +DMARDs therapy (experimental group) or DMARDs therapy (control group). Patients will undergo MSC transplant at the start of the study on Day 0. After 3 months, patients will receive the second MSC transplantation. After six and twelve months from the first transplantation, patients will be evaluated.
|Contact: chengyun zheng, Ph. Demail@example.com|
|Department of Hematology of the 2nd Hospital of Shandong University||Recruiting|
|Jinan, Shandong, China, 250033|
|Contact: chengyun zheng, Ph. D +86-531-85875635 firstname.lastname@example.org|
|Sub-Investigator: Ni Zhang|
|Principal Investigator:||chengyun zheng, Ph. D||Department of Hematology of The 2nd Hospital of Shandong University|