Systemic SclerosisPhase 1/2NCT00962923
Allogeneic Mesenchymal Stem Cells Transplantation for Systemic Sclerosis (SSc)
A Phase 1/2 study testing Allogeneic Mesenchymal Stem Cells (AlloMSC) in people with Systemic Sclerosis and Mesenchymal Stem Cells, run by The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School at 1 site in China.
Trial summary
This study will explore safety and efficacy of allogeneic mesenchymal stem cells transplantation (MSCT) to treat patients with diagnosis of systemic sclerosis(SSc) who have been resistant to multiple standard treatments. Read more about this study
- Who is it for?
- Ages 15 to 65 years
- Conditions the study is for
- Systemic Sclerosis, Mesenchymal Stem Cells
- What is being tested?
- Allogeneic Mesenchymal Stem Cells (AlloMSC)
- Type of treatment
- Injection or infusion
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Aug 2009to Dec 2011
- Phase
- Phase 1/2
- 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 15 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All patients fulfilled the American College of Rheumatology (former American Rheumatism Association - ARA) for SSc
- Rapidly progressive disease <2 years duration with a modified Rodnan skin score(mRSS) above 20, plus ESR >25 mm/first h and/or Hb <11 g/dL, not explained by other causes than active SSc
- lung involvement: with a vital capacity (VC) or DLCO below 70% predicted, or a mean pulmonary artery pressure (PAP) above 40 mmHg (measured by echocardiography)
- digestive tract involvement: with serum albumin ,25 g/L or weight loss exceeding 10% body weight in the preceding year
- kidney involvement: with 24-h urinary protein above 0.5 g or serum creatinine above 120 mmol/L
Who cannot join
- Uncontrolled arrhythmia, echocardiographic left ventricular ejection fraction (LVEF) <50% or mean PAP >50 mmHg, DLCO<45% of predicted
- Creatinine clearance <20 ml/min
- Platelets<80 000/mm3, haemorrhagic cystitis
- (4) HIV or HTLV1 seropositivity, malignancy, pregnancy, a cardiac or vascular prosthesis, and no vascular access
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
the Affiliated Drum Tower Hospital of Nanjing University Medical School
Nanjing, Jiangsu, 210008
Yitao Ding, MD, +86-25-83106666 ext. 66866, yitaoding@hotmail.com
About this study
This study will explore safety and efficacy of allogeneic mesenchymal stem cells transplantation (MSCT) to treat patients with diagnosis of systemic sclerosis(SSc) who have been resistant to multiple standard treatments. The underlying hypothesis is that the SSc condition is caused by an abnormal immune homeostasis that can be restored by MSCT.
Read the detailed description
- To test a new approach using allogeneic derived mesenchymal stem cell based therapy (MSCT) to treat refractory SSc
- To determine the disease-free survival in SSc patients treated with MSCT
- To assess adverse events of allogeneic MSC transplantation
- To assess the association of remission for organ function, clinical score and SSc serology levels at baseline with disease-free survival
- Conditions
- Systemic Sclerosis, Mesenchymal Stem Cells
- Being tested
- Allogeneic Mesenchymal Stem Cells (AlloMSC)
- Keywords
- Systemic sclerosis, Mesenchymal Stem Cells, Transplantation
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Mesenchymal Stem Cells (AlloMSC)Biological
Allogeneic mesenchymal stem cells will be infused intravenously (single dose, 10^6 cells/kg body weight).
What is being measured
The primary outcome is the main question the study is designed to answer.
mRSS score,HRQOL score, SF-36 score for SSc patients
Measured: monthly
Secondary outcomes (3)
Remission for organ function, VC, DLCO, PAP, serum albumin, serum creatitin, weight loss, 24h proteinuria
Measured: every three month
SSc Serology(ATA,ACA,ANA,anti-ssDNA,anti-dsDNA,IgM,IgG,and IgA,complement C3 and C4
Measured: every three month
Change of peripheral blood B and T cells
Measured: every three month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2009 (estimated)
- Study startAug 2009
- Primary completionDec 2010 (estimated)
- Study completionDec 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical SchoolThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00962923
- Sponsor's protocol ID
- NJGLYY003
- Status last verified
- Aug 2009
- First submitted
- 19 Aug 2009
- Last updated
- 20 Aug 2009
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sun L, Akiyama K, Zhang H, Yamaza T, Hou Y, Zhao S, Xu T, Le A, Shi S. Mesenchymal stem cell transplantation reverses multiorgan dysfunction in systemic lupus erythematosus mice and humans. Stem Cells. 2009 Jun;27(6):1421-32. doi: 10.1002/stem.68.
PubMed 19489103Liang J, Zhang H, Hua B, Wang H, Wang J, Han Z, Sun L. Allogeneic mesenchymal stem cells transplantation in treatment of multiple sclerosis. Mult Scler. 2009 May;15(5):644-6. doi: 10.1177/1352458509104590.
PubMed 19389752Zhou K, Zhang H, Jin O, Feng X, Yao G, Hou Y, Sun L. Transplantation of human bone marrow mesenchymal stem cell ameliorates the autoimmune pathogenesis in MRL/lpr mice. Cell Mol Immunol. 2008 Dec;5(6):417-24. doi: 10.1038/cmi.2008.52.
PubMed 19118507Sun LY, Zhang HY, Feng XB, Hou YY, Lu LW, Fan LM. Abnormality of bone marrow-derived mesenchymal stem cells in patients with systemic lupus erythematosus. Lupus. 2007;16(2):121-8. doi: 10.1177/0961203306075793.
PubMed 17402368Alip M, Wang D, Zhao S, Li S, Zhang D, Duan X, Wang S, Hua B, Wang H, Zhang H, Feng X, Sun L. Umbilical cord mesenchymal stem cells transplantation in patients with systemic sclerosis: a 5-year follow-up study. Clin Rheumatol. 2024 Mar;43(3):1073-1082. doi: 10.1007/s10067-024-06865-z. Epub 2024 Jan 11.
PubMed 38206544Zhang H, Liang J, Tang X, Wang D, Feng X, Wang F, Hua B, Wang H, Sun L. Sustained benefit from combined plasmapheresis and allogeneic mesenchymal stem cells transplantation therapy in systemic sclerosis. Arthritis Res Ther. 2017 Jul 19;19(1):165. doi: 10.1186/s13075-017-1373-2.
PubMed 28724445Akiyama K, Chen C, Wang D, Xu X, Qu C, Yamaza T, Cai T, Chen W, Sun L, Shi S. Mesenchymal-stem-cell-induced immunoregulation involves FAS-ligand-/FAS-mediated T cell apoptosis. Cell Stem Cell. 2012 May 4;10(5):544-55. doi: 10.1016/j.stem.2012.03.007. Epub 2012 Apr 26.
PubMed 22542159
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Mesenchymal Stem Cells Transplantation for Systemic Sclerosis (SSc)
- ClinicalTrials.gov ID
- NCT00962923
- Acronym / sponsor ID
- NJGLYY003
- Conditions
- Systemic Sclerosis, Mesenchymal Stem Cells
- Keywords
- Systemic sclerosis, Mesenchymal Stem Cells, Transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical SchoolUniversity, hospital or other
- Study start
- Aug 2009
- Primary completion
- Dec 2010 (estimated)
- Study completion
- Dec 2011 (estimated)
- First posted
- 20 Aug 2009 (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.
This study will explore safety and efficacy of allogeneic mesenchymal stem cells transplantation (MSCT) to treat patients with diagnosis of systemic sclerosis(SSc) who have been resistant to multiple standard treatments. The underlying hypothesis is that the SSc condition is caused by an abnormal immune homeostasis that can be restored by MSCT.
Detailed description
- To test a new approach using allogeneic derived mesenchymal stem cell based therapy (MSCT) to treat refractory SSc
- To determine the disease-free survival in SSc patients treated with MSCT
- To assess adverse events of allogeneic MSC transplantation
- To assess the association of remission for organ function, clinical score and SSc serology levels at baseline with disease-free survival
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 15 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- All patients fulfilled the American College of Rheumatology (former American Rheumatism Association - ARA) for SSc
- Rapidly progressive disease <2 years duration with a modified Rodnan skin score(mRSS) above 20, plus ESR >25 mm/first h and/or Hb <11 g/dL, not explained by other causes than active SSc
- lung involvement: with a vital capacity (VC) or DLCO below 70% predicted, or a mean pulmonary artery pressure (PAP) above 40 mmHg (measured by echocardiography)
- digestive tract involvement: with serum albumin ,25 g/L or weight loss exceeding 10% body weight in the preceding year
- kidney involvement: with 24-h urinary protein above 0.5 g or serum creatinine above 120 mmol/L
Key exclusion criteria (4 listed)
- Uncontrolled arrhythmia, echocardiographic left ventricular ejection fraction (LVEF) <50% or mean PAP >50 mmHg, DLCO<45% of predicted
- Creatinine clearance <20 ml/min
- Platelets<80 000/mm3, haemorrhagic cystitis
- (4) HIV or HTLV1 seropositivity, malignancy, pregnancy, a cardiac or vascular prosthesis, and no vascular access
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 |
|---|---|---|
| Allogeneic Mesenchymal Stem Cells (AlloMSC)Allogeneic mesenchymal stem cells will be infused intravenously (single dose, 10^6 cells/kg body weight). | Biological | – |
Primary outcome
mRSS score,HRQOL score, SF-36 score for SSc patients
Time frame: monthly
3 secondary outcomes
Remission for organ function, VC, DLCO, PAP, serum albumin, serum creatitin, weight loss, 24h proteinuria
Time frame: every three month
SSc Serology(ATA,ACA,ANA,anti-ssDNA,anti-dsDNA,IgM,IgG,and IgA,complement C3 and C4
Time frame: every three month
Change of peripheral blood B and T cells
Time frame: every three month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2009.
- Background: PubMed 19489103Sun L, Akiyama K, Zhang H, Yamaza T, Hou Y, Zhao S, Xu T, Le A, Shi S. Mesenchymal stem cell transplantation reverses multiorgan dysfunction in systemic lupus erythematosus mice and humans. Stem Cells. 2009 Jun;27(6):1421-32. doi: 10.1002/stem.68.
- Background: PubMed 19389752Liang J, Zhang H, Hua B, Wang H, Wang J, Han Z, Sun L. Allogeneic mesenchymal stem cells transplantation in treatment of multiple sclerosis. Mult Scler. 2009 May;15(5):644-6. doi: 10.1177/1352458509104590.
- Background: PubMed 19118507Zhou K, Zhang H, Jin O, Feng X, Yao G, Hou Y, Sun L. Transplantation of human bone marrow mesenchymal stem cell ameliorates the autoimmune pathogenesis in MRL/lpr mice. Cell Mol Immunol. 2008 Dec;5(6):417-24. doi: 10.1038/cmi.2008.52.
- Background: PubMed 17402368Sun LY, Zhang HY, Feng XB, Hou YY, Lu LW, Fan LM. Abnormality of bone marrow-derived mesenchymal stem cells in patients with systemic lupus erythematosus. Lupus. 2007;16(2):121-8. doi: 10.1177/0961203306075793.
- Derived from this study: PubMed 38206544Alip M, Wang D, Zhao S, Li S, Zhang D, Duan X, Wang S, Hua B, Wang H, Zhang H, Feng X, Sun L. Umbilical cord mesenchymal stem cells transplantation in patients with systemic sclerosis: a 5-year follow-up study. Clin Rheumatol. 2024 Mar;43(3):1073-1082. doi: 10.1007/s10067-024-06865-z. Epub 2024 Jan 11.
- Derived from this study: PubMed 28724445Zhang H, Liang J, Tang X, Wang D, Feng X, Wang F, Hua B, Wang H, Sun L. Sustained benefit from combined plasmapheresis and allogeneic mesenchymal stem cells transplantation therapy in systemic sclerosis. Arthritis Res Ther. 2017 Jul 19;19(1):165. doi: 10.1186/s13075-017-1373-2.
- Derived from this study: PubMed 22542159Akiyama K, Chen C, Wang D, Xu X, Qu C, Yamaza T, Cai T, Chen W, Sun L, Shi S. Mesenchymal-stem-cell-induced immunoregulation involves FAS-ligand-/FAS-mediated T cell apoptosis. Cell Stem Cell. 2012 May 4;10(5):544-55. doi: 10.1016/j.stem.2012.03.007. Epub 2012 Apr 26.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Allogeneic Mesenchymal Stem Cells (AlloMSC) in people with Systemic Sclerosis and Mesenchymal Stem Cells, run by The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School 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 5 inclusion and 4 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 20 Aug 2009. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Lingyun Sun, MD (+86-25-83105219, lingyunsun2001@yahoo.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
mRSS score,HRQOL score, SF-36 score for SSc patients (time frame: monthly). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00962923, which also lists 7 linked publications.
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 20 Aug 2009.