Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

NCT03171194 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 00061632
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

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).

Conditions

System; Lupus Erythematosus

Keywords

Lupus; Stem Cell; Systemic Lupus Erythematosus

Outcome Measures

Primary Outcomes (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.

  Week 24

Secondary Outcomes (8)

• Frequency of All Adverse Events

  Baseline to Week 52

• Change in Disease Activity

  Baseline to Week 24

• Change in Patient Reported Outcomes - Life

  Baseline to Week 24

• Change in Patient Reported Outcomes - Fatigue

  Baseline to Week 24

• Change in Patient Reported Outcomes - Pain

  Baseline to Week 24

Study Arms (1)

Drug: Low Dose Mesenchymal Stem Cells ( MSCs)

EXPERIMENTAL

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.

Drug: Low Dose Mesenchymal Stem Cells (MSCs)

Interventions

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.

Drug: Low Dose Mesenchymal Stem Cells ( MSCs)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Active CNS lupus affecting mental status
• Active lupus nephritis requiring dialysis
• 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

Sponsors & Collaborators

• Medical University of South Carolina: lead

Study Sites (2)

Emory University

Atlanta, Georgia, 30322, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Related Publications (1)

• 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.

  PMID: 35820718 DERIVED

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Diane L. Kamen, MD, MSCR

  Medical University of South Carolina

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2017
• First Posted: May 31, 2017
• Study Start: April 27, 2017
• Primary Completion: April 30, 2018
• Study Completion: October 25, 2018
• Last Updated: May 2, 2019

Record last verified: 2019-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)