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Systemic Lupus ErythematosusPhase 1NCT02741362

Safety and Efficacy of Adipose Derived Stem Cells in Refractory Rheumatoid Arthritis, Systemic Lupus Erythematosus or Sharp's Syndrome

A Phase 1 study testing Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCs and Lipoaspiration in people with Systemic Lupus Erythematosus and Rheumatoid Arthritis, run by Arkansas Heart Hospital at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a Phase I-II open- label single-dose study in subjects with significant refractory Rheumatoid Arthritis (RA), relapsing Systemic Lupus Erythematosus (SLE) or Sharp's Syndrome (SS). This study will enroll a minimum of 20 subjects for RA, 20 subjects for SLE and 20 patients for SS. Read more about this study

Who is it for?
Ages 18 to 99 years
What is being tested?
Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCs and Lipoaspiration
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
2 participants
Where
1 siteUnited States
Study dates
Dec 2015to 31 Dec 2016
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 99 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Males and Females ≥18 years
  • Cognitive competitiveness.
  • Diagnosis of refractory RA (patient's poor response of 3 months of DMARDs determined and documented by a rheumatologist) ,diagnosis of relapsing SLE (more than 2 episodes in a year during optimal medical SLE therapy) or diagnosis of Sharp syndrome (Alarcon-Segovia's criteria: patients with U1 Ribonucleic protein (RNP) ≥ 1:1600, and three of the following: Swollen hands, Synovitis, Myositis, Raynauld's phenomenon, Acrosclerosis)
  • Life expectancy greater than 8 months
  • Ability to understand and provide signed informed consent, or have a designated legal guardian or spouse legally able and willing to make such decisions on the subject's behalf
  • Reasonable expectation that patient will receive standard post-treatment care and attend all scheduled safety follow-up visits
  • No changes in active pulmonary medications for heart failure during the two weeks prior enrollment.
  • Written informed consent

Who cannot join

  • Currently on systemic steroids (if chronic steroid therapy cannot be stopped, the patient will qualify if he/she is on a minimum maintenance dose per SLE, RA or SS guidelines of only one systemic steroid and constituting a bioequivalent to normal endogenous levels of that hormone).
  • Dementia.
  • Presence of immune deficiency.
  • Currently on NSAIDs, TNFa inhibitors, Calcium channel blockers or P2Y12 inhibitors
  • Complicated type 2 diabetes mellitus (presence of diabetic foot, advanced kidney disease or retinopathy).
  • History of cancer within the past 5 years.
Show all 14Show fewer
  • Presence of any other clinically-significant medical condition, psychiatric condition, or laboratory abnormality, that in the judgment of the Investigator or Sponsor for which participation in the study would pose a safety risk to the subject
  • Participation in another study with an investigational drug or device within 3 weeks prior to treatment
  • History within the past year of drug or alcohol abuse.
  • Females known to be pregnant, lactating or having a positive pregnancy test (will be tested during screening) or planning to become pregnant during the study.
  • Inability to comply with the conditions of the protocol.
  • Porphyria.
  • Allergy to sodium citrate or any "caine" type of local anesthetic.
  • Patient scheduled for hospice care.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Arkansas Heart Hospital

    Little Rock, Arkansas, 72211

Other trials in United States

About this study

Official title: A Phase I-II Open- Label Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Autologous Adipose Stem Cell (Adult Human) to Subjects With Refractory Rheumatoid Arthritis, Relapsing Systemic Lupus Erythematosus or Sharp's Syndrome

This is a Phase I-II open- label single-dose study in subjects with significant refractory Rheumatoid Arthritis (RA), relapsing Systemic Lupus Erythematosus (SLE) or Sharp's Syndrome (SS).

This study will enroll a minimum of 20 subjects for RA, 20 subjects for SLE and 20 patients for SS. 6 week data of serum Tumor Necrosis Factor- alpha (TNFa), Interleukin- 6 (IL-6), C- Reactive Protein (CRP), Erythrocyte Sedimentation Rate (ESR), Cluster of Differentiation (CD)4 +CD25 + Forkhead box P3(Foxp3) + regulatory T cells, Disease Activity Score for 28 joints (DAS-28) score and pain score will be collected in all patients who are enrolled in the study for the RA group (Baseline and 6 weeks after). For the SLE group, Transforming Growth Factor- beta (TGF-β), TNFa, IL-6, Interleukin- 17 (IL-17), CD3+CD8-IL17A+ T helper-17 (Th17) cells, CD4+CD25+Foxp3+ regulatory T cells and the Systemic Lupus Erythematosus Quality of Life Questionnaire (SLEQoL) score will be collected in all the subjects of this group. SS group will undergo the assessments of RA and SLE. Prior to the stem cell treatment, the patient will be assessed for 6 weeks by all the previously mentioned markers. Then, patients will receive the infusion of stromal vascular fraction cells containing the adult adipose derived stem cells 'aADSC' (single intravenous dose). The disease- modifying anti-rheumatic drugs (DMARDs) or the standard SLE treatment will not be interrupted with the exception of systemic steroids (excluding minimal maintenance dose of one steroid) during the duration of the study. Follow up visits will take place at 6 weeks, 3 Months and 6 Months after the cell infusion. Safety will be monitored on an ongoing basis, and an interim safety review will be conducted by the Investigator(s) and Sponsor after the first 10 patients have been enrolled and treated in each group.

Being tested
Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCs, Lipoaspiration
Keywords
Type III hypersensitivity autoimmune disease
Medical subject headings
Lupus Erythematosus, Systemic, Arthritis, Rheumatoid, Mixed Connective Tissue Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCsOther

    Also called Tissue transplant

  • LipoaspirationOther

Study groups in NCT02741362
Study groupRoleWhat they receive
ADSC armExperimentalSingle intravenous administration of Stromal Vacsular Fraction (SVF) cells soinating Adipose Derived Stem Cells (ADSC) 6 week baseline data prior to the injection of ADSC will be collected. 6 week, 3 months and 6 months follow up data will be compared against baseline.
Receives: Other: Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCs; Other: Lipoaspiration

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Frequency of Adverse Events (AE) and Serious Adverse Events (SAE) during a 6 month follow up period.

    Measured: 6 Months

Secondary outcomes (10)
  1. Quality of Life Questionnaires for SLE/ RA

    SLEQoL score

    Measured: 6 Months

  2. Quality of Life Questionnaires for RA

    DAS-28 score

    Measured: 6 Months

  3. Blood Flow Cytometry for CD4+CD25+Foxp3+ regulatory T cells

    CD4+CD25+Foxp3+ regulatory T cells,

    Measured: 6 Months

  4. Blood Flow Cytometry for CD3+CD8-IL17A+TH17 cells

    CD3+CD8-IL17A+TH17 cells

    Measured: 6 Months

  5. Serum C- reactive protein (CRP)

    C- reactive protein (CRP)

    Measured: 6 Months

  6. Erythrocyte Sedimentation Rate (ESR)

    Erythrocyte Sedimentation Rate (ESR)

    Measured: 6 Months

  7. Serum Tumor Necrosis Factor- alpha (TNFa)

    Tumor Necrosis Factor- alpha (TNFa)

    Measured: 6 Months

  8. Serum Transforming Growth Factor- beta (TGFb)

    Transforming Growth Factor- beta (TGFb)

    Measured: 6 Months

  9. Serum Interleukin- 6 (IL-6)

    Interleukin- 6 (IL-6)

    Measured: 6 Months

  10. Serum Interleukin- 17 (IL-17)

    Interleukin- 17 (IL-17)

    Measured: 6 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Apr 2016 (estimated)
  2. Study startDec 2015
  3. Primary completion31 Dec 2016
  4. Study completion31 Dec 2016

Why it stopped: Primary Investigator no longer with institution

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
2 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02741362
Sponsor's protocol ID
ICMS-2015-51
Status last verified
Mar 2017
First submitted
6 Apr 2016
Last updated
3 Mar 2017
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Wang D, Huang S, Yuan X, Liang J, Xu R, Yao G, Feng X, Sun L. The regulation of the Treg/Th17 balance by mesenchymal stem cells in human systemic lupus erythematosus. Cell Mol Immunol. 2017 May;14(5):423-431. doi: 10.1038/cmi.2015.89. Epub 2015 Oct 5.

    PubMed 26435067
  • Sharp GC, Irvin WS, Tan EM, Gould RG, Holman HR. Mixed connective tissue disease--an apparently distinct rheumatic disease syndrome associated with a specific antibody to an extractable nuclear antigen (ENA). Am J Med. 1972 Feb;52(2):148-59. doi: 10.1016/0002-9343(72)90064-2. No abstract available.

    PubMed 4621694
  • Deng W, Chen W, Zhang Z, Huang S, Kong W, Sun Y, Tang X, Yao G, Feng X, Chen W, Sun L. Mesenchymal stem cells promote CD206 expression and phagocytic activity of macrophages through IL-6 in systemic lupus erythematosus. Clin Immunol. 2015 Dec;161(2):209-16. doi: 10.1016/j.clim.2015.07.011. Epub 2015 Jul 22.

    PubMed 26209923
  • Garimella MG, Kour S, Piprode V, Mittal M, Kumar A, Rani L, Pote ST, Mishra GC, Chattopadhyay N, Wani MR. Adipose-Derived Mesenchymal Stem Cells Prevent Systemic Bone Loss in Collagen-Induced Arthritis. J Immunol. 2015 Dec 1;195(11):5136-48. doi: 10.4049/jimmunol.1500332. Epub 2015 Nov 4.

    PubMed 26538398
  • Kamata Y, Iwamoto M, Muroi K, Minota S. Repeated local implantation of autologous peripheral blood mononuclear cells for the treatment of ischaemic digits in patients with connective tissue diseases. Rheumatology (Oxford). 2011 May;50(5):906-10. doi: 10.1093/rheumatology/keq389. Epub 2010 Dec 15.

    PubMed 21169196

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Intravenous injection of Stromal Vascular Fraction Cells (SVF) containing ADSCs and Lipoaspiration in people with Systemic Lupus Erythematosus and Rheumatoid Arthritis, run by Arkansas Heart Hospital at 1 site in United States.

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 8 inclusion and 14 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: stopped early, as of the sponsor's last update on 3 Mar 2017. 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 United States. 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?

The study was stopped early (reason given: Primary Investigator no longer with institution). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency of Adverse Events (AE) and Serious Adverse Events (SAE) during a 6 month follow up period. (time frame: 6 Months). 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 NCT02741362, which also lists 5 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 3 Mar 2017.

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