Clinical trial NCT01420432: Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Ankylosing Spondylitis (AS)
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat AS
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Ankylosing Spondylitis
Keywords: Ankylosing Spondylitis
Umbilical Cord/placenta-Derived MSC
Transplantation
Lead sponsor: Shandong University
Sponsor class: OTHER
First posted: 2011-08-19
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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)

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Patient age 18\~60 years old with plan to infuse MSCs.
2. Diagnosis of "Definite AS" (arthritis of the spine) as defined by the modified New York criteria
3. Stable doses of sulfasalazine,methotrexate,thalidomide,hydroxychloroquine, low-dose corticosteroids, and NSAIDs are permitted
4. Patients must have an ECOG 0\~2.
5. No moderate or sever organ dysfunction: Ejection fraction\>45%; Creatinine \<176 umol/L.
6. No severe infection.
7. Each patient must sign written informed consent.

Exclusion Criteria:

1. Other serious concomitant diseases (uncontrolled/severe kidney, liver, haematological, gastrointestinal, endocrine, cardiovascular, pulmonary, neurological or cerebral disease)
2. Psychiatric condition that would limit informed consent.
3. HIV, hepatitis B or C, tuberculosis, other infections
4. Positive Pregnancy Test or lactation
5. Patient has enrolled another clinical trial study within last 4 weeks.
6. Contraindications to MSC

Primary outcomes:
measure: The Assessment of Spondyloarthritis International Society (ASAS)20 response
timeFrame: 1 year
measure: erythrocyte sedimentation rate (ESR)
timeFrame: 1 year
measure: imageology
timeFrame: 1 year
measure: C-reactive protein (CRP)
timeFrame: 1 year
Secondary outcomes:
measure: Percentage of systemic T regulatory cell population
timeFrame: 1 year
measure: Side effects
timeFrame: 1 year
Other outcomes:
Locations:
city: Jinan
country: China
facility: Department of Hematology of the 2nd Hospital of Shandong University
state: Shandong
Study officials:
affiliation: Department of Hematology of The 2nd Hospital of Shandong University
name: chengyun zheng, Ph. D
role: PRINCIPAL_INVESTIGATOR
References: