Phase 2 Trial of Mesenchymal Stem Cells in Systemic Lupus Erythematosus (MiSLE)
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|ClinicalTrials.gov Identifier: NCT02633163|
Recruitment Status : Recruiting
First Posted : December 17, 2015
Last Update Posted : April 1, 2019
|Condition or disease||Intervention/treatment||Phase|
|Systemic Lupus Erythematosus||Drug: Low Dose Mesenchymal Stem Cells (MSCs) Drug: High Dose Mesenchymal Stem Cells (MSCs) Drug: Placebo Infusion||Phase 2|
A phase 2 multicenter (several medical research centers participating), placebo controlled, randomized (assigned by chance), double blind (neither the participant nor the investigator will know if active drug or placebo is assigned) trial to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) for the treatment of systemic lupus erythematosus (SLE) in adults.
The MSCs will be obtained from healthy donor umbilical cords and two doses of MSCs will be tested. The cells will be produced at the Medical University of South Carolina (MUSC) and will be shipped to other participating centers for patients with SLE. Participants will receive either active drug or placebo through a single IV infusion. All participants will receive standard of care and their safety will be monitored throughout the study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||81 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase II Controlled Trial of Allogeneic Mesenchymal Stem Cells for the Treatment of Refractory Lupus|
|Actual Study Start Date :||October 26, 2018|
|Estimated Primary Completion Date :||June 2021|
|Estimated Study Completion Date :||June 2021|
Experimental: Low Dose Mesenchymal Stem Cells (MSCs)
Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution
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.
Experimental: High Dose Mesenchymal Stem Cells (MSCs)
Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution
Drug: High Dose Mesenchymal Stem Cells (MSCs)
Participants will receive a single IV infusion of Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution.
Placebo Comparator: Plasma Lyte A Solution
Placebo Infusion (Plasma-Lyte A solution only)
Drug: Placebo Infusion
Participants will receive a placebo infusion that does not contain any mesenchymal stem cells.The placebo infusion will consist of Plasma-Lyte A, which is the same vehicle used to deliver the MSCs in the experimental groups.
- Clinical response at Week 24 as defined by the SLE Responder Index (SRI): [ Time Frame: Week 24 ]
Systemic Lupus Erythematosus Responder Index (SRI) is defined as a greater than or equal to (≥) 4 point reduction in the Systemic Lupus Erythematosus Disease Activity Index score (SLEDAI), no new British Isles Lupus Assessment Group (BILAG) A or no more than 1 new BILAG B domain score, and no deterioration from Baseline in the Physician's Global Assessment (PGA) by greater than or equal to (≥) 0.3 points.
Additionally, to be a "responder", corticosteroid dose must be less than of equal to (≤)10 mg/day of prednisone or equivalent by the Week 20 visit and be maintained at less than or equal to 10 mg/day through Week 24.
- Change in SLEDAI score between groups [ Time Frame: Baseline to Weeks 12, 24, and 52 ]Changes in Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) between the experimental and placebo group.
- Renal and non-renal organ system flares [ Time Frame: At or before Weeks 12, 24, and 52 ]Frequency of renal and non-renal organ system flares at or before Weeks 12, 24, and 52, defined by the BILAG criteria.
- Changes in SLICC-DI [ Time Frame: Baseline to Week 52 ]• Change in SLICC-DI from Baseline to Week 52 to assess for accumulation of new damage (SLE-related or treatment-related)
- Changes in HR-QOL [ Time Frame: Baseline to Week 52 ]Changes in HR-QOL (measured by SF36 v2) from Baseline to Weeks 12, 24, and 52
- Changes in Fatigue [ Time Frame: Baseline to Week 52 ]Changes in fatigue (measured by PROMIS Fatigue Short Form (SF)) from Baseline to Weeks 12, 24, and 52
- Changes in Pain [ Time Frame: Baseline to Week 52 ]Changes in pain (measured by PROMIS Pain SF) from Baseline to Weeks 12, 24, and 52
- Changes in Depression [ Time Frame: Baseline to Week 52 ]Changes in depression (measured by PROMIS Depression SF) from Baseline to Weeks 12, 24, and 52
- Changes in patient-reported lupus-specific disease status [ Time Frame: Baseline to Week 52 ]Changes in patient-reported lupus-specific disease status (measured by the LupusPro and LIT) from Baseline to Weeks 12, 24, and 52
- Steroid-sparing effect [ Time Frame: Baseline to Week 52 ]Steroid-sparing effect (measured by discontinuation of corticosteroids and time to discontinuation among those taking corticosteroids)
- Cumulative systemic steroid dose [ Time Frame: Week 52 ]Cumulative systemic steroid dose (PO, IV, IM) at Week 52
- Changes in the presence of serum and urine biomarkers of SLE activity: [ Time Frame: Baseline to Week 52 ]Changes in the presence of serum and urine biomarkers of SLE activity: SLE-related cytopenias, low serum complement levels, anti-dsDNA levels or urine protein measures from Baseline to Weeks 12, 24, and 52.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02633163
|Contact: Gary Gilkeson||(843) 792 - email@example.com|
|United States, California|
|Cedars-Sinai Medical Center||Recruiting|
|Los Angeles, California, United States, 90048|
|Contact: Bonnie Paul 310-423-1232 firstname.lastname@example.org|
|Principal Investigator: Mariko L. Ishimori, MD|
|Sub-Investigator: Daniel J. Wallace, MD|
|University of California - San Diego||Recruiting|
|San Diego, California, United States, 92093|
|Contact: Kenneth Kalunian, MD 858-246-2381 email@example.com|
|Principal Investigator: Kenneth Kalunian, MD|
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|Contact: Karla Caylor, RN 404-616-7553 firstname.lastname@example.org|
|Principal Investigator: S. Sam Lim, MD, MPH|
|Sub-Investigator: Arezou Khosroshahi, MD|
|United States, Illinois|
|Chicago, Illinois, United States, 60611|
|Contact: Karen Mancera-Cuevas, MS, MPH 312-503-0251 email@example.com|
|Principal Investigator: Rosalind Ramsey-Goldman, MD|
|Sub-Investigator: Cybele Ghossein, MD|
|United States, New York|
|University of Rochester Medical Center||Recruiting|
|Rochester, New York, United States, 14642|
|Contact: Maria Allen 585-275-7167 Maria_Allen@urmc.Rochester.edu|
|Principal Investigator: Ummara Shah, MD|
|Sub-Investigator: R. John Looney, MD|
|United States, North Carolina|
|University of North Carolina at Chapel Hill||Recruiting|
|Chapel Hill, North Carolina, United States, 27599|
|Contact: Brenda Meier, RN 919-445-2730 firstname.lastname@example.org|
|Principal Investigator: Saira Z Sheikh, MD|
|United States, South Carolina|
|Medical University of South Carolina||Recruiting|
|Charleston, South Carolina, United States, 29425|
|Contact: Gary Gilkeson, MD 843-792-6043 email@example.com|
|Contact: Angela R Brown, MA 843-792-6043 firstname.lastname@example.org|
|Principal Investigator: Gary S. Gilkeson, MD|
|Sub-Investigator: Diane L. Kamen, MD, MSCR|
|Principal Investigator:||Gary S. Gilkeson, MD||Medical University of South Carolina|
|Study Chair:||Diane L. Kamen, MD, MSCR||Medical University of South Carolina|