C-CAR168 CAR T Cell Therapy for Refractory Autoimmune Disease

Launched by ABELZETA INC. · Apr 11, 2025

Keywords

ClinConnect Summary

The C-CAR168 clinical trial is studying a new type of treatment called CAR T-cell therapy for patients with lupus nephritis (LN), a serious autoimmune disease that can affect the kidneys. This trial is designed for people whose lupus has not responded to standard treatments. The main goals are to see if the treatment is safe, how well patients can tolerate it, and to find the best dose for future studies. Participants will receive a single infusion of the therapy after undergoing a process to collect their own immune cells, which will be modified and then re-infused to help fight their disease.

To be eligible for this trial, participants must be between 18 and 70 years old and have a confirmed diagnosis of lupus nephritis that has not improved with at least two treatments. Participants will need to stop certain medications before starting the trial and will be monitored closely for safety and effectiveness for up to 24 months after receiving the treatment. This study is not yet recruiting patients, but it offers a potential new option for those struggling with refractory autoimmune diseases like lupus nephritis.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Informed Consent: Voluntary signed consent required.
• • 2. Age \& Gender: Males and females, 18-70 years old.
• • 3. Diagnosis: Clinical diagnosis of SLE per EULAR/ACR criteria for at least 6 months.
• • 4. Lupus Nephritis (LN): Biopsy-confirmed active proliferative LN (Class III/IV ± V) within the past 12 months.
• 5. Refractory Disease:
• • Treated with at least two immunosuppressants for ≥8 weeks.
• • Stable but active disease despite standard therapy (steroids, IS, monoclonal antibodies).
• • Steroid dose ≤30 mg/day (if applicable).
• 6. Disease Activity at Screening:
• • SLE without LN: SLEDAI-2K ≥8 and 1 BILAG A or 2 BILAG B scores.
• • LN Patients: Proteinuria ≥1.0 g/day or UPCR ≥1.0 g/g.
• 7. Autoantibody Status:
• • o Positive ANA (≥1:80), anti-dsDNA (≥30 IU/mL), and/or anti-Smith antibody.
• • 8. Infection Status: No active infection within 2 weeks before leukapheresis.
• • 9. Life Expectancy: Greater than 6 months.
• 10. Adequate Organ Function:
• • Bone Marrow: ANC ≥1.0×10⁹/L, ALC ≥0.5×10⁹/L, Hb ≥80 g/L, PLT ≥75×10⁹/L.
• • Coagulation: INR/APTT ≤1.5×ULN.
• • Cardiac: LVEF ≥45% by ECHO/MUGA.
• • Pulmonary: SpO₂ ≥92% on room air.
• • Liver: ALT/AST ≤2.5×ULN, total bilirubin \<2.0 mg/dL.
• • Renal: Creatinine clearance ≥40 mL/min (Cockcroft-Gault).
• 11. Pregnancy \& Contraception:
• • Women of childbearing potential must have a negative pregnancy test at screening.
• • Both male and female participants must use highly effective contraception for 1 year post-treatment.
• Exclusion Criteria:
• • 1. Any other concomitant diseases requiring long term systemic steroids (oral or intravenously) treatment that may confound the interpretation of study results or have interference with background steroid tapering for the subjects.
• 2. Any of the following:
• • Positive for Hepatitis B surface antigen (HBsAg)/core antibody (HBcAb)/e antibody (HBeAb)/e antigen (HBeAg).
• • Positive for Hepatitis C Virus (HCV) antibodies.
• • Positive for Human Immunodeficiency Virus (HIV) antibodies.
• • Positive for syphilis antigen or antibody.
• • 3. Have an uncontrolled active infection.
• • 4. History of major organ transplantation (such as heart, lung, liver, kidney) or history of bone marrow/hematopoietic stem cell transplantation.
• • 5. History of any of stroke, unstable angina, myocardial infarction, congestive heart failure (NYHA Class III or IV), severe cardiomyopathy or ventricular arrhythmia requiring medication or mechanical control within 6 months of screening.
• • 6. History of ≥ Grade 2 bleeding within the past 30 days.
• • 7. Received a live vaccine within 4 weeks prior to signing the ICF.
• 8. Received any of the following treatments:
• • Prednisone treatment of ≥ 100 mg/d or equivalent corticosteroid therapy for ≥14 days within the previous 8 weeks.
• • Receive plasma exchange, plasma separation, hemodialysis, or intravenous injection of immunoglobulin (IVIG) within 14 days prior to leukapheresis.
• • Use of any other investigational clinical study drug within 28 days prior to leukapheresis. However, if the subject is not responsive to the treatment or have progressed and at least 3 half-lives have passed before the leukapheresis, he/she could be enrolled.
• • Previously received any CAR-T cell products or other genetically modified T cell therapies.
• • Rituximab/ocrelizumab/obinutuzumab within 6 months prior to screening
• • 9. Pregnant or breastfeeding women.
• • 10. History of seizure disorder, cerebrovascular ischemia/hemorrhage, dementia or cerebellar disease or other severe neuropsychiatric syndromes.
• • 11. History of deep vein thrombosis or pulmonary embolism within six months of infusion (line associated DVT is allowed)
• • 12. Diagnosed with malignant tumors within 5 years prior to signing the ICF, with the following exceptions: non-melanoma skin cancer that has been treated with radical therapy, localized prostate cancer, biopsy-confirmed cervical carcinoma in situ or squamous intraepithelial lesions detected by cervical smear, and completely excised breast carcinoma in situ.
• • 13. Poor compliance, unwilling or unable to adhere to the study protocol based on the investigator's assessment.
• • 14. Allergies to fludarabine, cyclophosphamide and/or known allergies to excipients of C-CAR168 cell product.