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System; Lupus ErythematosusPhase 1NCT03171194

Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

A Phase 1 study testing Low Dose Mesenchymal Stem Cells (MSCs) in people with System; Lupus Erythematosus, run by Medical University of South Carolina at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to evaluate the safety of mesenchymal stromal cells (MSCs) obtained from umbilical cords for the treatment of adults with active systemic lupus erythematosus (SLE). Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
System; Lupus Erythematosus
What is being tested?
Low Dose Mesenchymal Stem Cells (MSCs)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
2 sitesUnited States
Study dates
27 Apr 2017to 25 Oct 2018
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 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients between 18 and 65 years old, male or female, of any race
  • Definite SLE by meeting either SLICC or ACR Classification Criteria for SLE
  • Evidence of a positive ANA (≥1:80 titer) or positive dsDNA antibody test within 6 months of screening
  • Clinically mild to moderately active SLE determined by SLEDAI score ≥4 and ≤10 at screening, despite SOC therapy
  • If the patient has BILAG A or two BILAG Bs in the renal organ system, he/she must have completed at least 6 months of therapy with either mycophenolate mofetil or cyclophosphamide for the current episode of nephritis
  • Able and willing to give written informed consent

Who cannot join

  • Active CNS lupus affecting mental status
  • Active lupus nephritis requiring dialysis
  • Laboratory exclusions: eGFR <30, WBC <2.0/mm3, hemoglobin <8 g/dL, platelet count <30,000/mm3, liver enzymes AST or ALT >4 times upper limit normal; Positive testing for HIV, hepatitis B or hepatitis C
  • History of malignant neoplasm within the last 3 years, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix
  • Pregnant or breast feeding; males or females not willing to use adequate contraception
  • History of renal transplantation
Show all 14Show fewer
  • Herpes zoster within the past 90 days or any infection requiring hospitalization or intravenous antibiotics within the past 60 days
  • Clinically significant EKG or chest X-ray abnormalities
  • Any other medical condition, related or unrelated to SLE, that in the opinion of the investigator would render the patient inappropriate or too unstable to complete study protocol
  • Use of prednisone >0.5 mg/kg/day (or equivalent corticosteroid) within 1 month of Baseline visit
  • Change or addition to immunosuppressant regimen within 3 months of Baseline visit (except corticosteroids); Use of other experimental therapeutic agents within 3 months of Baseline visit
  • Having received belimumab within 3 months of Baseline, or having received rituximab or other B cell depleting biologic therapy within 6 months of Baseline.
  • Comorbidities requiring corticosteroid therapy
  • Current substance abuse or recent (within 60 days) history of substance abuse

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

Other trials in United States

About this study

Official title: A Phase I Safety Trial of Allogeneic Mesenchymal Stem Cells for Systemic Lupus Erythematosus

The purpose of this study is to evaluate the safety of mesenchymal stromal cells (MSCs) obtained from umbilical cords for the treatment of adults with active systemic lupus erythematosus (SLE).

Read the detailed description

This open label trial will evaluate the safety of allogeneic MSCs for the treatment of adults with moderate to severely active systemic lupus erythematosus (SLE). MSCs will be derived from healthy donor umbilical cord cells and 1 dose of MSCs will be tested. MUSC has a good manufacturing practice (GMP) quality Clean Cell Facility to ensure the quality and safety of the MSCs prior to infusing into study participants. The goal of this study is to determine the safety of MSC infusion in patients with SLE when added to standard of care for SLE.

The MSCs used in this trial are cells that are obtained from the umbilical cords of healthy donors having an elective Caesarean section and who have been screened to be sure that they are free of any infectious diseases. These investigational cells will be collected and processed so that they can be used as an infusion treatment. An infusion is when a drug (in this case the MSCs) is administered directly into the blood stream via a vein, usually located in the arm or hand. All participants will receive standard of care and their safety will be monitored throughout the study.

Being tested
Low Dose Mesenchymal Stem Cells (MSCs)
Keywords
Lupus, Stem Cell, Systemic Lupus Erythematosus
Medical subject headings
Lupus Erythematosus, Systemic

What participants receive

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

  • Low Dose Mesenchymal Stem Cells (MSCs)Drug

    Mesenchymal stromal/stem cells (MSCs) are cells that can be derived from umbilical cords, bone marrow, adipose tissue, and dental pulp, among other sites. MSCs have the ability to mediate a range of immuno-modulatory actions for both the innate and adaptive immune systems.

Study groups in NCT03171194
Study groupRoleWhat they receive
Drug: Low Dose Mesenchymal Stem Cells ( MSCs)ExperimentalParticipants will receive a single IV infusion of Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution. All participants will receive the infusion at the Baseline (Day 0) visit. All participants will continue on their standard-of-care therapy during the trial.
Receives: Drug: Low Dose Mesenchymal Stem Cells (MSCs)

What is being measured

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

  1. Frequency of Grade 3 or higher adverse events

    The primary outcome measure is the frequency of Grade 3 or higher adverse events (AEs) experienced by participants at or prior to Week 24.

    Measured: Week 24

Secondary outcomes (8)

  1. Frequency of All Adverse Events

    Frequency of all adverse events (AEs) including any serious AEs (SAEs) at or prior to Week 52.

    Measured: Baseline to Week 52

  2. Change in Disease Activity

    Change in SLE disease activity between Baseline and Week 24 measured by change in SLEDAI score and change in prednisone dose.

    Measured: Baseline to Week 24

  3. Change in Patient Reported Outcomes - Life

    Changes between Baseline and Week 24 in patient-reported quality of life

    Measured: Baseline to Week 24

  4. Change in Patient Reported Outcomes - Fatigue

    Changes between Baseline and Week 24 in patient-reported measures of fatigue.

    Measured: Baseline to Week 24

  5. Change in Patient Reported Outcomes - Pain

    Changes between Baseline and Week 24 in patient-reported measures of pain.

    Measured: Baseline to Week 24

  6. Change in Patient Reported Outcomes - Depression

    Changes between Baseline and Week 24 in patient-reported measures of depression.

    Measured: Baseline to Week 24

  7. Change in Disease Biomarkers - Cellular

    Changes between Baseline and Week 24 in cellular markers of inflammation and autoimmunity. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.

    Measured: Baseline to Week 24

  8. Change in Disease Biomarkers - Serum

    Changes between Baseline and Week 24 in serum markers of inflammation and autoimmunity. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.

    Measured: Baseline to Week 24

Timeline

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

  1. First posted31 May 2017
  2. Study start27 Apr 2017
  3. Primary completion30 Apr 2018
  4. Study completion25 Oct 2018

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
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03171194
Sponsor's protocol ID
00061632
Status last verified
Apr 2019
First submitted
4 May 2017
Last updated
2 May 2019
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Kamen DL, Wallace C, Li Z, Wyatt M, Paulos C, Wei C, Wang H, Wolf BJ, Nietert PJ, Gilkeson G. Safety, immunological effects and clinical response in a phase I trial of umbilical cord mesenchymal stromal cells in patients with treatment refractory SLE. Lupus Sci Med. 2022 Jul;9(1):e000704. doi: 10.1136/lupus-2022-000704.

    PubMed 35820718

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Low Dose Mesenchymal Stem Cells (MSCs) in people with System; Lupus Erythematosus, run by Medical University of South Carolina at 2 sites 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 6 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: finished, as of the sponsor's last update on 2 May 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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 Grade 3 or higher adverse events (time frame: Week 24). 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 NCT03171194, which also lists 1 linked publication.

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 2 May 2019.

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