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

Ankylosing Spondylitis Clinical Trial

Official title:

Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat AS

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01420432
• Other study ID #: Zhangni
• Status: Recruiting
• Phase: Phase 1
• First received: August 11, 2011
• Last updated: August 18, 2011
• Start date: January 2011
• Est. completion date: December 2013

Study information

• Verified date: April 2011
• Source: Shandong University
• Contact: chengyun zheng, Ph. D
• Phone: +86-531-85875635
• Email: chengyun.zheng[@]ki.se
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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)

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: December 2013
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ankylosing Spondylitis
• Spondylitis
• Spondylitis, Ankylosing

Intervention

Biological:
• Human umbilical cord-derived MSCs
1.0E+6 MSC/kg, IV drop and repeat repeated after three months

Locations

Country	Name	City	State
China	Department of Hematology of the 2nd Hospital of Shandong University	Jinan	Shandong

Sponsors (1)

Lead Sponsor	Collaborator
Shandong University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Assessment of Spondyloarthritis International Society (ASAS)20 response	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)	1 year	No
Primary	erythrocyte sedimentation rate (ESR)	erythrocyte sedimentation rate (ESR) level will be mainly observed after transplanting 3, 6,12-month.	1 year	No
Primary	imageology	imageology will be mainly observed after transplanting 3, 6,12-month.	1 year	No
Primary	C-reactive protein (CRP)	C-reactive protein (CRP) level will be mainly observed after transplanting 3, 6,12-month.	1 year	No
Secondary	Percentage of systemic T regulatory cell population	Percentages of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year	1 year	No
Secondary	Side effects	Side effects were observed after the treatment	1 year	No