Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

NCT ID: NCT03171194

Last Updated: 2019-05-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-04-27
• Study Completion Date: 2018-10-25

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.

Conditions

System; Lupus Erythematosus

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Drug: Low Dose Mesenchymal Stem Cells ( MSCs)

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.

Group Type: EXPERIMENTAL

Low Dose Mesenchymal Stem Cells (MSCs)

Intervention Type: 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.

Interventions

Low Dose Mesenchymal Stem Cells (MSCs)

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.

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Medical University of South Carolina

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Diane L. Kamen, MD, MSCR

Role: STUDY_CHAIR

Medical University of South Carolina

Locations

Emory University

Atlanta, Georgia, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 35820718 (View on PubMed)

Other Identifiers

00061632

Identifier Type: -

Identifier Source: org_study_id