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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 April 2021
• Main ID: NCT04835883
• Date of registration: 06/04/2021
• Prospective Registration: No
• Primary sponsor: Hanyang University Seoul Hospital
• Public title: Exploring the Efficacy and Safety of CS20AT04 (Allogenic Bone Marrow-Derived Stem Cell) in Systemic Lupus Erythematosus Patients
• Scientific title: 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
• Date of first enrolment: September 26, 2019
• Target sample size: 10
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT04835883
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Korea, Republic of

Contacts

• Name: Chan-Bum Choi, M.D.,Ph.D

• Name: Chan-Bum Choi, M.D.,PhD.

• Name: Chan-Bum Choi, M.D.,Ph.D.
• Affiliation: Department of Rhumatology in Hanyang University Medical Center

Key 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Lupus Erythematosus

Intervention(s)

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

Primary Outcome(s)

Reduction of corticosteroid dose to = 7.5 mg/day of prednisone or equivalent [Time Frame: 24 weeks]

Hematologic improvement without ongoing SLE treatment [Time Frame: 24 weeks]

Renal response improvement [Time Frame: 24 weeks]

Secondary Outcome(s)

Proportion of subjects with baseline Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) = 6 having SLE responder index 6 (SRI6) [Time Frame: 24 weeks]

Change from baseline of hematologic parameters [Time Frame: 3 days, 1 week, 4 weeks, 12 weeks , and 24 weeks]

Proportion of subjects achieving Low Level Disease Activity State [Time Frame: 24 weeks]

Change in baseline of renal function parameters [Time Frame: 24 weeks]

Time to first renal flare [Time Frame: 24 weeks]

Proportion of subjects who achieve a prednisone dose of = 7.5mg/day or equivalent [Time Frame: 12 weeks, 24 weeks]

Proportion of subjects with baseline Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) = 5 having SLE responder index 5 (SRI5) [Time Frame: 24 weeks]

Time to first severe modified SLE Flare Index (SFI) flare [Time Frame: 24 weeks]

Time to event for each of the individual components of treatment failure [Time Frame: 24 weeks]

Time to complete remission [Time Frame: 24 weeks]

Proportion of subjects with = 4 point reduction from baseline in Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) score [Time Frame: 24 weeks]

Time to first hematologic flare [Time Frame: 24 weeks]

Proportion of subjects with baseline Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) = 4 having SLE responder index 4 (SRI5) [Time Frame: 24 weeks]

Secondary ID(s)

CS20AT04-SLE101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Corestem, Inc.

Ministry of Health & Welfare, Korea