Ankylosing SpondylitisPhase 1NCT01420432
Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Ankylosing Spondylitis (AS)
A Phase 1 study testing Human umbilical cord-derived MSCs in people with Ankylosing Spondylitis, run by Shandong University at 1 site in China.
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) Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Ankylosing Spondylitis
- What is being tested?
- Human umbilical cord-derived MSCs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- Jan 2011to Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patient age 18~60 years old with plan to infuse MSCs.
- Diagnosis of "Definite AS" (arthritis of the spine) as defined by the modified New York criteria
- Stable doses of sulfasalazine,methotrexate,thalidomide,hydroxychloroquine, low-dose corticosteroids, and NSAIDs are permitted
- Patients must have an ECOG 0~2.
- No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.
- No severe infection.
- Each patient must sign written informed consent.
Who cannot join
- Other serious concomitant diseases (uncontrolled/severe kidney, liver, haematological, gastrointestinal, endocrine, cardiovascular, pulmonary, neurological or cerebral disease)
- Psychiatric condition that would limit informed consent.
- HIV, hepatitis B or C, tuberculosis, other infections
- Positive Pregnancy Test or lactation
- Patient has enrolled another clinical trial study within last 4 weeks.
- Contraindications to MSC
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology of the 2nd Hospital of Shandong University
Jinan, Shandong, 250033
chengyun zheng, Ph. D, +86-531-85875635, chengyun.zheng@ki.se
Ni Zhang
About this study
Official title: Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat 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)
Read the 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.
- Conditions
- Ankylosing Spondylitis
- Being tested
- Human umbilical cord-derived MSCs
- Keywords
- Ankylosing Spondylitis, Umbilical Cord/placenta-Derived MSC, Transplantation
- Medical subject headings
- Spondylitis, Ankylosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human umbilical cord-derived MSCsBiological
1.0E+6 MSC/kg, IV drop and repeat repeated after three months
| Study group | Role | What they receive |
|---|---|---|
| Human umbilical cord-derived MSCs and DMARDs | Experimental | 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 Receives: Biological: Human umbilical cord-derived MSCs |
| DMARDs | No intervention | DMARDs such as sulfasalazine,methotrexate,thalidomide po for 12 months |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 1 year
erythrocyte sedimentation rate (ESR)
erythrocyte sedimentation rate (ESR) level will be mainly observed after transplanting 3, 6,12-month.
Measured: 1 year
imageology
imageology will be mainly observed after transplanting 3, 6,12-month.
Measured: 1 year
C-reactive protein (CRP)
C-reactive protein (CRP) level will be mainly observed after transplanting 3, 6,12-month.
Measured: 1 year
Secondary outcomes (2)
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
Measured: 1 year
Side effects
Side effects were observed after the treatment
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2011 (estimated)
- Study startJan 2011
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ankylosing SpondylitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shandong University
- Sponsor type
- University, hospital or other
- Investigators
- chengyun zheng, Ph. D, Principal investigator, Department of Hematology of The 2nd Hospital of Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01420432
- Sponsor's protocol ID
- Zhangni
- Status last verified
- Apr 2011
- First submitted
- 11 Aug 2011
- Last updated
- 19 Aug 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat AS
- ClinicalTrials.gov ID
- NCT01420432
- Acronym / sponsor ID
- Zhangni
- Conditions
- Ankylosing Spondylitis
- Keywords
- Ankylosing Spondylitis, Umbilical Cord/placenta-Derived MSC, Transplantation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Shandong UniversityUniversity, hospital or other
- Study start
- Jan 2011
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 19 Aug 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Human umbilical cord-derived MSCs and DMARDs | Experimental | Biological: Human umbilical cord-derived MSCsHuman 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 |
| DMARDs | No intervention | –DMARDs such as sulfasalazine,methotrexate,thalidomide po for 12 months |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Patient age 18~60 years old with plan to infuse MSCs.
- Diagnosis of "Definite AS" (arthritis of the spine) as defined by the modified New York criteria
- Stable doses of sulfasalazine,methotrexate,thalidomide,hydroxychloroquine, low-dose corticosteroids, and NSAIDs are permitted
- Patients must have an ECOG 0~2.
- No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.
Key exclusion criteria (6 listed)
- Other serious concomitant diseases (uncontrolled/severe kidney, liver, haematological, gastrointestinal, endocrine, cardiovascular, pulmonary, neurological or cerebral disease)
- Psychiatric condition that would limit informed consent.
- HIV, hepatitis B or C, tuberculosis, other infections
- Positive Pregnancy Test or lactation
- Patient has enrolled another clinical trial study within last 4 weeks.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human umbilical cord-derived MSCs1.0E+6 MSC/kg, IV drop and repeat repeated after three months | Biological | Human umbilical cord-derived MSCs and DMARDs |
Primary outcome
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)
erythrocyte sedimentation rate (ESR)
Time frame: 1 year
erythrocyte sedimentation rate (ESR) level will be mainly observed after transplanting 3, 6,12-month.
imageology
Time frame: 1 year
imageology will be mainly observed after transplanting 3, 6,12-month.
C-reactive protein (CRP)
Time frame: 1 year
C-reactive protein (CRP) level will be mainly observed after transplanting 3, 6,12-month.
2 secondary outcomes
Percentage of systemic T regulatory cell population
Time frame: 1 year
Side effects
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
chengyun zheng, Ph. D principal investigator
Department of Hematology of The 2nd Hospital of Shandong University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Human umbilical cord-derived MSCs in people with Ankylosing Spondylitis, run by Shandong University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact chengyun zheng, Ph. D (+86-531-85875635, chengyun.zheng@ki.se), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Assessment of Spondyloarthritis International Society (ASAS)20 response (time frame: 1 year). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2011.