Clinical trial NCT03171194: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: System; Lupus Erythematosus
Keywords: Lupus
Stem Cell
Systemic Lupus Erythematosus
Lead sponsor: Medical University of South Carolina
Sponsor class: OTHER
First posted: 2017-05-31
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Frequency of Grade 3 or higher adverse events
timeFrame: Week 24
Secondary outcomes:
measure: Frequency of All Adverse Events
timeFrame: Baseline to Week 52
measure: Change in Disease Activity
timeFrame: Baseline to Week 24
measure: Change in Patient Reported Outcomes - Life
timeFrame: Baseline to Week 24
measure: Change in Patient Reported Outcomes - Fatigue
timeFrame: Baseline to Week 24
measure: Change in Patient Reported Outcomes - Pain
timeFrame: Baseline to Week 24
measure: Change in Patient Reported Outcomes - Depression
timeFrame: Baseline to Week 24
measure: Change in Disease Biomarkers - Cellular
timeFrame: Baseline to Week 24
measure: Change in Disease Biomarkers - Serum
timeFrame: Baseline to Week 24
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: Medical University of South Carolina
name: Diane L. Kamen, MD, MSCR
role: STUDY_CHAIR
References:
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.