Exploring the Efficacy and Safety of CS20AT04 (Allogenic Bone Marrow-Derived Stem Cell) in Systemic Lupus Erythematosus Patients

Launched by HANYANG UNIVERSITY SEOUL HOSPITAL · Apr 6, 2021

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called CS20AT04, which uses special stem cells derived from bone marrow to help patients with systemic lupus erythematosus (SLE). Specifically, the study focuses on two groups: those with lupus nephritis (kidney inflammation due to lupus) and those with lupus cytopenia (low blood cell counts). The trial aims to determine how effective and safe this treatment is for these patients.

To participate, patients must have a compatible bone marrow donor and meet certain criteria, such as having a positive test for specific lupus-related antibodies and not responding well to standard treatments. Participants in the trial can expect to receive this new therapy while being closely monitored for their health and any side effects. It’s important to know that the trial is currently recruiting patients, and those interested should discuss it with their healthcare provider to see if they qualify.

Gender

ALL

Eligibility 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