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

Who is this study for? Patients with Systemic Lupus Erythematosus
What treatments are being studied? CS20AT04 (Allogenic Bone Marrow Derived Mesenchymal Stem Cell)
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This is an open-label, one-arm single-center phase Ⅱa study exploring the efficacy and safety of CS20AT04 (HLA-haplo Matched Allogenic Bone Marrow-Derived Stem Cells) in two subpopulation group of systemic lupus erythematosus patients - lupus nephritis and lupus cytopenia.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: f
View:

• Patients with HLA-haplo-matched bone marrow donor less than 70 years old

• 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

• 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

• 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)

Locations
Other Locations
Republic of Korea
Hanyang University Medical Center
RECRUITING
Seoul
Contact Information
Primary
Chan-Bum Choi, M.D.,Ph.D
cbchoi@hanyang.ac.kr
+82222909208
Backup
Kwijoo Kim
kwjkim@corestem.com
+821027568321
Time Frame
Start Date: 2019-09-26
Estimated Completion Date: 2026-01-20
Participants
Target number of participants: 10
Treatments
Experimental: Assigned interventions
Subjects enrolled into the CS20AT04 with corticosteroid taper regimen arm will receive two infusions of CS20AT04 (2.0×10\^6cell/kg), on 0 day and on 12 weeks post-enrollment.
Sponsors
Collaborators: Corestemchemon, Inc., Ministry of Health & Welfare, Korea
Leads: Hanyang University Seoul Hospital

This content was sourced from clinicaltrials.gov