Efficacy and Safety of CRC01 in Participants With Severe, Refractory Systemic Lupus Erythematosus

NCT07364396 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: CRC01-02
• Study Type: interventional
• Target: 39
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this clinical trial is to evaluate the safety and efficacy of CRC01, an investigational autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, in people with lupus nephritis (LN), a serious kidney complication of systemic lupus erythematosus (SLE). The main objectives of the study are:

• Undergo eligibility screening, including blood tests, urine tests, and disease activity assessments.
• Provide autologous T lymphocytes through a procedure called leukapheresis.
• Receive a lymphodepleting pre-conditioning regimen (short course of chemotherapy).
• Receive a single intravenous infusion of CRC01 cells.
• Be hospitalized for close monitoring to detect and manage early adverse events such as CRS or neurotoxicity.
• Return for scheduled follow-up assessments through Week 52 (12 months) post-infusion to evaluate safety and treatment response. Key Outcomes Researchers will measure:
• Changes in proteinuria and kidney function.
• Changes in disease activity scores.
• Incidence and severity of adverse events.

Conditions

Lupus Nephritis (LN); SLE; SLE (Systemic Lupus); Lupus Nephritis

Outcome Measures

Primary Outcomes (6)

• Phase 1 Study: To evaluate the tolerability of CRC01 in patients with severe refractory autoimmune disease (systemic lupus erythematosus), and to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D)

  Safety and tolerability will be assessed by: 1\. Number of participants with dose-limiting toxicities (DLTs) within 28 days after CRC01 infusion, as assessed by CTCAE v5.0 and ASTCT consensus criteria.

  28 day

• Phase 1 Study: To evaluate the tolerability of CRC01 in patients with severe refractory autoimmune disease (systemic lupus erythematosus), and to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D)

  Safety and tolerability will be assessed by: 2\. Determination of the maximum tolerated dose (MTD) defined as the highest dose level at which ≤1 of 6 participants experience a DLT.

  28 day

• Phase 1 Study: To evaluate the tolerability of CRC01 in patients with severe refractory autoimmune disease (systemic lupus erythematosus), and to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D)

  Safety and tolerability will be assessed by: 3\. Determination of the recommended Phase 2 dose (RP2D) based on incidence of DLTs, safety profile, and investigator/sponsor assessment.

  28 day

• Phase 2 Study: To evaluate the efficacy of CRC01 by assessing Complete Renal Response (CRR)

  Complete Renal Response (CRR): Defined as meeting all of the following criteria: 1\. 24-hour urine protein ≤0.5 g, or urine protein-to-creatinine ratio (UPCR) ≤0.5. If a 24-hour urine collection is available and meets adequacy criteria, 24-hour urine protein assessment takes precedence. If the 24-hour urine collection is inadequate or not performed, UPCR will be used for evaluation.

  24 weeks

• Phase 2 Study: To evaluate the efficacy of CRC01 by assessing Complete Renal Response (CRR)

  Complete Renal Response (CRR): Defined as meeting all of the following criteria: 2\. eGFR ≥60 mL/min/1.73m², or a decrease in eGFR ≤20% compared with the pre-flare value.

  24 weeks

• Phase 2 Study: To evaluate the efficacy of CRC01 by assessing Complete Renal Response (CRR)

  Complete Renal Response (CRR): Defined as meeting all of the following criteria: 3\. No use of rescue medication for systemic lupus erythematosus (SLE). (Use of corticosteroids equivalent to ≤7.5 mg/day prednisone is permitted.)

  24 weeks

Secondary Outcomes (13)

• Complete Renal Response (CRR) or Partial Renal Response (PRR) at each assessment Visit

  Up to Week 52

• Change From Baseline in Urine Protein-to-Creatinine Ratio (UPCR)

  up to Week 52

• Change From Baseline in Serum Creatinine

  up to Week 52

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

  up to Week 52

• Change from baseline in UPCR, serum creatinine, urine protein, and eGFR

  up to Week 52

Study Arms (1)

CRC01

EXPERIMENTAL

Participants with lupus nephritis will receive a single intravenous infusion of CRC01 (autologous anti-CD19 CAR-T cells) following lymphodepleting pre-conditioning chemotherapy.

Biological: CRC01

Interventions

CRC01 BIOLOGICAL

Autologous T lymphocytes genetically modified to express anti-CD19 chimeric antigen receptor (CAR). Administered as a single intravenous infusion.

CRC01

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 19 years or older, voluntarily provides written informed consent.
• Diagnosis of systemic lupus erythematosus (SLE) according to the 2019 EULAR/ACR classification criteria.
• Positive antinuclear antibody (ANA) at screening (titer ≥1:80).
• Diagnosis of lupus nephritis Class III or IV (with or without concurrent Class V), confirmed by kidney biopsy within 1 year prior to screening based on ISN/RPS 2018 criteria.
• Inadequate response to, or intolerance of, at least two or more standard therapies for 6 months or longer (cyclophosphamide, mycophenolate mofetil, azathioprine, tacrolimus, rituximab, belimumab).
• Proteinuria at screening with urine protein-to-creatinine ratio (UPCR) \>1.5.
• Adequate laboratory values at screening: Hemoglobin \>8.0 g/dL; ANC \>1,000/μL; Platelets ≥50,000/μL; Total bilirubin ≤2.0 × ULN; AST and ALT ≤3 × ULN; eGFR ≥30 mL/min/1.73 m².
• Hemodynamically stable, no pericardial effusion, and LVEF ≥50% by echocardiogram at screening.
• FEV1/FVC ≥70% at screening.
• Willing and able to comply with study visits, procedures, and requirements.
• Women of childbearing potential and men must agree to use effective contraception for at least 1 year after CRC01 infusion until PCR testing confirms clearance of CRC01.

You may not qualify if:

• Current or anticipated requirement for renal dialysis during the study.
• History of kidney transplantation or planned transplantation during the study.
• History of severe CNS lupus or currently active severe CNS lupus.
• Prior CAR-T cell therapy.
• History of malignancy except for: basal or squamous cell carcinoma of skin treated and disease-free ≥3 years; in-situ carcinoma of cervix or breast treated and disease-free ≥3 years; superficial bladder cancer treated and disease-free ≥3 years; completely resected primary malignancy in complete remission ≥5 years.
• Unstable angina and/or myocardial infarction within 1 year prior to screening.
• Congestive heart failure of NYHA Class III or IV within 1 year prior to screening.
• Thromboembolism, pulmonary embolism, or clinically significant bleeding diathesis within 6 months prior to screening.
• Hypoxemia, clinically significant pleural effusion, or abnormal ECG findings within 6 months prior to screening.
• Stroke (ischemic or hemorrhagic) within 6 months prior to screening.
• Positive HBsAg; positive anti-HCV (eligible if HCV RNA negative); known HIV infection; or active neurological autoimmune/inflammatory diseases (e.g., Guillain-Barré syndrome, ALS).
• Recurrent or symptomatic ventricular tachycardia, or atrial fibrillation with rapid ventricular response despite therapy within 3 months prior to screening.
• Severe or uncontrolled active infection requiring systemic therapy at screening.
• Rapidly progressive disease or otherwise unsuitable for study participation, per investigator judgment.
• Pregnant or breastfeeding women.

Sponsors & Collaborators

• Curocell Inc.: lead

MeSH Terms

Conditions

Lupus Nephritis; Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Sihong Choi

CONTACT

+821033848516; sihong.choi@curocellbtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 20, 2025
• First Posted: January 23, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): June 1, 2030
• Study Completion (Estimated): June 1, 2030
• Last Updated: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share