Using stem cells to treat systemic lupus erythematosus
Open-label, Single-arm, 24-week Investigator Study to Evaluate the Efficacy and Safety of CS20AT04 (HLA-haplo Matched Allogenic Bone Marrow-Derived Stem Cells) Administered in Patients With Lupus Nephritis or Lupus Cytopenia
This study is testing a new stem cell treatment to see if it can help people with systemic lupus erythematosus, especially those with kidney issues and low blood cell counts, feel better and stay in remission.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Hanyang University Seoul Hospital Academic / other |
| Drugs / interventions | cyclophosphamide, prednisone |
| Locations | 1 site (Seoul) |
| Trial ID | NCT04835883 on ClinicalTrials.gov |
What this trial studies
This phase 2 clinical trial investigates the efficacy and safety of CS20AT04, an allogenic bone marrow-derived stem cell therapy, in patients with systemic lupus erythematosus (SLE), specifically targeting those with lupus nephritis and lupus cytopenia. The study is open-label and single-arm, meaning all participants will receive the treatment without a control group. Participants will receive two infusions of CS20AT04, with the first on the day of enrollment and the second 12 weeks later, while also tapering corticosteroid use. The goal is to assess the treatment's ability to induce and maintain remission in these patients who have not responded adequately to standard therapies.
Who should consider this trial
Good fit: Ideal candidates include patients with HLA-haplo-matched bone marrow donors who are under 70 years old and have a confirmed diagnosis of systemic lupus erythematosus.
Not a fit: Patients who do not have a suitable HLA-haplo-matched donor or those who are not treatment-refractory may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new effective therapy for patients with treatment-refractory systemic lupus erythematosus.
How similar studies have performed: While the use of stem cells in treating autoimmune diseases is being explored, this specific approach in systemic lupus erythematosus is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Patients with HLA-haplo-matched bone marrow donor less than 70 years old 2. Patients meeting: -at least 4 of the 2012 Systemic Lupus International Collaborating Clinics (SLICC) criteria, including at least 1 clinical criterion and 1 immunology criterion; or -at least 4 of the 11 Revised American College of Rheumatology (ACR) Criteria for Classification of Systemic Lupus Erythematosus, according to the 1997 Update of the 1982 ACR 3. Patients having a positive test result for antinuclear antibody (ANA; titer at least 1:80) and/or anti-double stranded DNA antibody (anti-dsDNA Ab) at screening 4. Patients (non-responder or partial responder), defined as : -unresponsive to treatment with standard care(such as monthly i.v. pulse cyclophosphamide (CYC) 500-1000 mg/m2, mycophenolate (MMF) ≥ 2 gm/day, azathioprine (AZA) ≥ 200 mg/day, leflunomide (LEF) 20 mg/day, oral CYC, cyclosporine, mizoribine ≥ 150 mg/day, mycophenolic acid ≥ 1.44 g/day, tacrolimus (TAC) ≥ 1.5 mg twice a day alone or in combination for at least 6 months) or -with continued daily dosage of ≥15mg of prednisone or its equivalent for maintenance treatment 5-1. For the lupus cytopenia sub-group only: * Patients with refractory cytopenia (at least one of anemia, leukopenia, or thrombocytopenia) in absence of any other identifiable cause, defined as: \[Red blood cell associated\] -Hemolytic anemia (Hgb ≤ 10g/dL) with reticulocytosis, or \[White cell associated\] -Neutrophil count \< 1,000/mm3 (in the absence of other known cause such as corticosteroids, drugs, and infection), and/or * Lymphocyte count \< 1,500/mm3 \[Platelet associated\] * Platelet count \< 100,000/mm3 (in the absence of other known cause such as drugs, portal hypertension, and thrombotic thrombocytopenic purpura (TTP)) 5-2. For the lupus nephritis sub-group only: •Patients with clinical disease activity of lupus nephritis, defined by: * laboratory tests documented active lupus nephritis three consecutive times: (i) decrease in renal function (serum creatinine \> 106 μmol/L) (ii) increase in proteinuria (defined as urine protein/creatinine ratio (UPC) \> 1), and (iii) deterioration in microscopic hematuria (defined as \> 10 red cells per high power field) in the absence of menstrual hematuria or urinary tract infection at the time of screening or the presence of cellular casts * renal biopsy documenting lupus nephritis according to the International Society of Nephrology/Renal Pathology Society classification of active or active/chronic lupus nephritis in renal biopsy class III, class IV-S or IV-G, class V, class III + V, or class IV + V (within 1 year) Exclusion Criteria: 1. Patients unable or unwilling to provide written informed consent 2. Patients with any history of cancer, allergy, alcohol or substance abuse, active peptic ulcer disease, heart failure, liver disease, and coagulation disorder 3. Patients who have active severe central nervous system (CNS) lupus 4. Patients who have received biologic investigational agents in the past year 5. Patients undergoing intravenous immunoglobulin or plasma exchange therapy 6. Patients who are pregnant or are lactating 7. Patients with any evidence of a major infection 8. For the lupus nephritis sub-group only: Patients with serum creatinine \> 250 μmol/L
Where this trial is running
Seoul
- Hanyang University Medical Center — Seoul, Korea, Republic of (Recruiting)
Study contacts
- Principal investigator: Chan-Bum Choi, M.D.,Ph.D. — Department of Rhumatology in Hanyang University Medical Center
- Study coordinator: Chan-Bum Choi, M.D.,Ph.D
- Email: cbchoi@hanyang.ac.kr
- Phone: +82222909208
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.