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

NCT04835883 | Unknown Phase 2

• 1 other identifier: CS20AT04-SLE101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Systemic Lupus Erythematosus

Keywords

Systemic lupus erythematosus; Allogenic bone marrow-derived mesenchymal stem cell; Lupus Nephritis; Lupus Cytopenia; Cell Therapy; Phase IIa Clinical Trial

Outcome Measures

Primary Outcomes (3)

• Reduction of corticosteroid dose to ≤ 7.5 mg/day of prednisone or equivalent

  The primary efficacy objective of this study is to assess the efficacy of CS20AT04, in combination with a 24-week corticosteroid taper regimen, as measured by: Reduction of corticosteroid dose to ≤ 7.5 mg/day of prednisone or equivalent at Week 4 , and continued through Week 12

  24 weeks

• Hematologic improvement without ongoing SLE treatment

  As Lupus cytopenia sub-group specific endpoint, Hgb increase of ≥ 1.5 g/dL, or Neutrophil count increase of ≥ 100% from pre-treatment level and an absolute increase of 500/mm3 x 10\^9/L, or For pre-treatment platelet count \> 20 X 10\^9/L, a platelet absolute increase of ≥ 30 X 10\^9/L For pre-treatment platelet count ≤ 20 X 10\^9/L, a platelet absolute increase ≥ 20 X 10\^9/L and ≥ 100% increase from pre-treatment level

  24 weeks

• Renal response improvement

  As Lupus nephritis (LN) sub-group specific endpoint, a renal response at 24 weeks adjudicated based on the following parameters: Urine Protein to Creatinine Ratio (UPCR) of ≤0.5mg/mg, and Estimated Glomerular Filtration Rate (eGFR)≥60mL/min/1.73m2 or no confirmed decrease from baseline in eGFR of \> 20%, and Did not receive any rescue medication for LN, and Did not receive over than 7.5mg prednisone for ≥7 consecutive days in total during week 12 through 24, just prior to the renal response assessment

  24 weeks

Secondary Outcomes (13)

• Proportion of subjects who achieve a prednisone dose of ≤ 7.5mg/day or equivalent

  12 weeks, 24 weeks

• Proportion of subjects achieving Low Level Disease Activity State

  24 weeks

• Proportion of subjects with ≥ 4 point reduction from baseline in Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) score

  24 weeks

• Proportion of subjects with baseline Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) ≥ 4 having SLE responder index 4 (SRI5)

  24 weeks

• Proportion of subjects with baseline Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) ≥ 5 having SLE responder index 5 (SRI5)

  24 weeks

Other Outcomes (2)

• Change in SLE parameters compare to baseline

  3 days, 1 week, 4 weeks, 12 weeks, and 24 weeks

• Change from baseline of Treg CD4+CD25+Foxp3+) and Th17 (CD3+CD8-IL-17A+) cell counts

  3 days, 1 week, 4 weeks, 12 weeks, 24 weeks and 0.5 years, 1 year, 1.5 years, 2 years, 3 years

Study Arms (1)

Assigned interventions

EXPERIMENTAL

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.

Biological: CS20AT04 (allogenic bone marrow derived mesenchymal stem cell)

Interventions

CS20AT04 (allogenic bone marrow derived mesenchymal stem cell) BIOLOGICAL

Two intravenous infusions of CS20AT04 (2.0×10\^6cell/kg), on 0 day and on 12 weeks The target population of this study is subjects in South Korea with a diagnosis of either lupus nephritis or lupus cytopenia.

Assigned interventions

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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))
• For the lupus nephritis sub-group only: •Patients with clinical disease activity of lupus nephritis, defined by:

You may not qualify if:

• \. Patients unable or unwilling to provide written informed consent
• \. Patients with any history of cancer, allergy, alcohol or substance abuse, active peptic ulcer disease, heart failure, liver disease, and coagulation disorder
• \. Patients who have active severe central nervous system (CNS) lupus
• \. Patients who have received biologic investigational agents in the past year
• \. Patients undergoing intravenous immunoglobulin or plasma exchange therapy
• \. Patients who are pregnant or are lactating
• \. Patients with any evidence of a major infection
• \. For the lupus nephritis sub-group only: Patients with serum creatinine \> 250 μmol/L

Sponsors & Collaborators

• Hanyang University Seoul Hospital: lead
• Corestemchemon, Inc.: collaborator
• Ministry of Health & Welfare, Korea: collaborator

Study Sites (1)

Hanyang University Medical Center

Seoul, KS013, South Korea

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Lupus Nephritis

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Officials

• Chan-Bum Choi, M.D.,Ph.D.

  Department of Rhumatology in Hanyang University Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chan-Bum Choi, M.D.,Ph.D

CONTACT

+82222909208; cbchoi@hanyang.ac.kr

Kwijoo Kim

CONTACT

+821027568321; kwjkim@corestem.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 6, 2021
• First Posted: April 8, 2021
• Study Start: September 26, 2019
• Primary Completion: November 5, 2021
• Study Completion: January 20, 2026
• Last Updated: April 8, 2021

Record last verified: 2021-04

Locations

KR (1)