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Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Ankylosing Spondylitis (AS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01420432
Recruitment Status : Unknown
Verified April 2011 by Shandong University.
Recruitment status was:  Recruiting
First Posted : August 19, 2011
Last Update Posted : August 19, 2011
Information provided by:
Shandong University

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)

Condition or disease Intervention/treatment Phase
Ankylosing Spondylitis Biological: Human umbilical cord-derived MSCs Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat AS
Study Start Date : January 2011
Estimated Primary Completion Date : December 2013
Estimated Study Completion Date : December 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. The Assessment of Spondyloarthritis International Society (ASAS)20 response [ Time Frame: 1 year ]

    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)

  2. erythrocyte sedimentation rate (ESR) [ Time Frame: 1 year ]
    erythrocyte sedimentation rate (ESR) level will be mainly observed after transplanting 3, 6,12-month.

  3. imageology [ Time Frame: 1 year ]
    imageology will be mainly observed after transplanting 3, 6,12-month.

  4. C-reactive protein (CRP) [ Time Frame: 1 year ]
    C-reactive protein (CRP) level will be mainly observed after transplanting 3, 6,12-month.

Secondary Outcome Measures :
  1. Percentage of systemic T regulatory cell population [ Time Frame: 1 year ]
    Percentages of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year

  2. Side effects [ Time Frame: 1 year ]
    Side effects were observed after the treatment

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01420432

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Contact: chengyun zheng, Ph. D +86-531-85875635

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China, Shandong
Department of Hematology of the 2nd Hospital of Shandong University Recruiting
Jinan, Shandong, China, 250033
Contact: chengyun zheng, Ph. D    +86-531-85875635   
Sub-Investigator: Ni Zhang         
Sponsors and Collaborators
Shandong University
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Principal Investigator: chengyun zheng, Ph. D Department of Hematology of The 2nd Hospital of Shandong University
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Responsible Party: Chengyun Zheng, Department of Hematology of the 2nd Hospital of Shandong University Identifier: NCT01420432    
Other Study ID Numbers: Zhangni
First Posted: August 19, 2011    Key Record Dates
Last Update Posted: August 19, 2011
Last Verified: April 2011
Keywords provided by Shandong University:
Ankylosing Spondylitis
Umbilical Cord/placenta-Derived MSC
Additional relevant MeSH terms:
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Spondylitis, Ankylosing
Bone Diseases, Infectious
Bone Diseases
Musculoskeletal Diseases
Spinal Diseases
Joint Diseases