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Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

Primary Purpose

System; Lupus Erythematosus

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Low Dose Mesenchymal Stem Cells (MSCs)

About this trial

This is an interventional treatment trial for System; Lupus Erythematosus focused on measuring Lupus, Stem Cell, Systemic Lupus Erythematosus

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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
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

Sites / Locations

Emory University
Medical University of South Carolina

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Drug: Low Dose Mesenchymal Stem Cells ( MSCs)

Arm Description

Participants 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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Frequency of All Adverse Events

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

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.

Change in Patient Reported Outcomes - Life

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

Change in Patient Reported Outcomes - Fatigue

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

Change in Patient Reported Outcomes - Pain

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

Change in Patient Reported Outcomes - Depression

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

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.

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.

Full Information

NCT ID

NCT03171194

First Posted

May 4, 2017

Last Updated

April 30, 2019

Sponsor

Medical University of South Carolina

1. Study Identification

Unique Protocol Identification Number

NCT03171194

Brief Title

Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2019

Overall Recruitment Status

Completed

Study Start Date

April 27, 2017 (Actual)

Primary Completion Date

April 30, 2018 (Actual)

Study Completion Date

October 25, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Medical University of South Carolina

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief 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).

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

System; Lupus Erythematosus

Keywords

Lupus, Stem Cell, Systemic Lupus Erythematosus

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Drug: Low Dose Mesenchymal Stem Cells ( MSCs)

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Low Dose Mesenchymal Stem Cells (MSCs)

Intervention Description

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.

Primary Outcome Measure Information:

Title

Frequency of Grade 3 or higher adverse events

Description

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

Time Frame

Week 24

Secondary Outcome Measure Information:

Title

Frequency of All Adverse Events

Description

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

Time Frame

Baseline to Week 52

Title

Change in Disease Activity

Description

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

Time Frame

Baseline to Week 24

Title

Change in Patient Reported Outcomes - Life

Description

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

Time Frame

Baseline to Week 24

Title

Change in Patient Reported Outcomes - Fatigue

Description

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

Time Frame

Baseline to Week 24

Title

Change in Patient Reported Outcomes - Pain

Description

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

Time Frame

Baseline to Week 24

Title

Change in Patient Reported Outcomes - Depression

Description

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

Time Frame

Baseline to Week 24

Title

Change in Disease Biomarkers - Cellular

Description

Time Frame

Baseline to Week 24

Title

Change in Disease Biomarkers - Serum

Description

Time Frame

Baseline to Week 24

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Diane L. Kamen, MD, MSCR

Organizational Affiliation

Medical University of South Carolina

Official's Role

Study Chair

Facility Information:

Facility Name

Emory University

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30322

Country

United States

Facility Name

Medical University of South Carolina

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29425

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

35820718

Citation

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.

Results Reference

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Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

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