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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2011 by Shandong University.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Shandong University
ClinicalTrials.gov Identifier:
NCT01420432
First received: August 11, 2011
Last updated: August 18, 2011
Last verified: April 2011
  Purpose

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 Intervention Phase
Ankylosing Spondylitis
Biological: Human umbilical cord-derived MSCs
Phase 1

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Shandong University:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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


Estimated Enrollment: 10
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)
Arms Assigned Interventions
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

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

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01420432

Contacts
Contact: chengyun zheng, Ph. D +86-531-85875635 chengyun.zheng@ki.se

Locations
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    chengyun.zheng@ki.se   
Sub-Investigator: Ni Zhang         
Sponsors and Collaborators
Shandong University
Investigators
Principal Investigator: chengyun zheng, Ph. D Department of Hematology of The 2nd Hospital of Shandong University
  More Information

No publications provided

Responsible Party: Chengyun Zheng, Department of Hematology of the 2nd Hospital of Shandong University
ClinicalTrials.gov Identifier: NCT01420432     History of Changes
Other Study ID Numbers: Zhangni
Study First Received: August 11, 2011
Last Updated: August 18, 2011
Health Authority: China: Food and Drug Administration

Keywords provided by Shandong University:
Ankylosing Spondylitis
Umbilical Cord/placenta-Derived MSC
Transplantation

Additional relevant MeSH terms:
Spondylitis
Spondylitis, Ankylosing
Bone Diseases, Infectious
Infection
Bone Diseases
Musculoskeletal Diseases
Spinal Diseases
Spondylarthropathies
Spondylarthritis
Ankylosis
Joint Diseases
Arthritis

ClinicalTrials.gov processed this record on August 27, 2014