A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy

NCT06249438 | Recruiting Phase 1

• 1 other identifier: 1141-043(CAR-AID)
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is an investigator-initiated, multicenter, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with autoimmune diseases refractory to standard therapy

Conditions

Systemic Lupus Erythematosus (SLE); Immune-mediated Necrotizing Myopathy (IMNM); Neuromyelitis Optica Spectrum Disorders (NMOSD); Multiple Sclerosis (MS); Myasthenia Gravis; Systemic Sclerosis (SSc)

Keywords

CD20/BCMA-directed CAR-T cells

Outcome Measures

Primary Outcomes (2)

• Incidence of Adverse Events [Safety and Tolerability]

  Incidence of any adverse events (AEs), including dose limiting toxicities (DLTs)

  Throughout the first 24 months follow up period completion (3 years)，DLTs will be observed/collected throughout the 28 days post C-CAR168 infusion

• The subsequent recommended dose of C-CAR168 in patients with autoimmune diseases refractory to standard therapy

  Based on the assessment of dose-limiting toxicities (DLTs) rates and overall safety profile

  Throughout the first 24 months follow up period completion (3 years)

Secondary Outcomes (14)

• The proportion of subjects who achieved remission at 6 months (6M)

  Throughout the first 6 months follow up period completion (1.5 years)

• The proportion of subjects who achieved remission during the main study period

  Throughout the first 24 months follow up period completion (3 years)

• The proportion of subjects who experienced relapse during the main study period

  Throughout the first 24 months follow up period completion (3 years)

• Time to response (TTR)

  Throughout the first 24 months follow up period completion (3 years)

• Progression-free survival (PFS)

  Throughout the first 24 months follow up period completion (3 years)

Other Outcomes (6)

• Serum cytokines (including Interleukin (IL)-2, IL-4, IL-6, IL-10, Tumor Necrosis Factor (TNF)-α, Interferon (IFN)-γ) changes

  Throughout the first 24 months follow up period completion (3 years)

• Soluble BCMA changes in peripheral blood

  Throughout the first 24 months follow up period completion (3 years)

• RNA changes in peripheral blood

  Throughout the first 24 months follow up period completion (3 years)

Study Arms (1)

C-CAR168

EXPERIMENTAL

Autologous C-CAR168 administered by intravenous (IV) infusion

Biological: CD20/BCMA-directed CAR-T cells

Interventions

CD20/BCMA-directed CAR-T cells BIOLOGICAL

Autologous 2nd generation CD20/BCMA-directed CAR-T cells, single infusion intravenously

Also known as: C-CAR168

C-CAR168

Eligibility Criteria

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

You may qualify if:

• to 70 years old at the time of signing the Informed Consent Form (ICF).
• Diagnosed as SLE/Immune-Mediated Necrotizing Myopathy (IMNM)/Neuromyelitis Optica Spectrum Disorders (NMOSD)/Multiple Sclerosis (MS)/Myasthenia Gravis (MG)/Systemic Sclerosis (SSc) according to recognized diagnostic criteria for at least 6 months.
• Remains disease active or relapses after treatment with standard of care therapy for at least 8 weeks with the dose stable for more than 2 weeks; patients should have been treated with at least two immunosuppressants (including immunosuppressants, biologics, and disease-modifying drug (DMD) ).
• Adequate bone marrow, coagulation, cardiopulmonary, liver and renal function.

You may not qualify if:

• Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), Treponema Pallidum (TP) positive, Cytomegalovirus (CMV) DNA positive, Epstein-Barr Virus (EBV) DNA positive.
• Uncontrolled active infection.
• Live vaccine injection within 4 weeks prior to signing the ICF.
• Major organ transplantation history or bone marrow/hematopoietic stem cell transplantation history.
• Severe cardiovascular diseases within the past 6 months prior to screening.
• ≥ Grade 2 bleeding within the past 30 days prior to screening, or requiring long-term anticoagulants treatment.
• Inadequate washing time for previous treatment.
• Previously treated with CAR-T cell products or genetically modified T cell therapies.
• Pregnant or lactating women.
• Severe central nervous system diseases or pathological changes.
• Malignancy history within 5 years prior to signing the ICF.

Sponsors & Collaborators

• RenJi Hospital: lead
• AbelZeta Pharma Inc.: collaborator

Study Sites (1)

Department of Rheumatology, RenJi Hospital, School of Medicine, Shanghai JiaoTong University

Shanghai, Shanghai Municipality, 200001, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Neuromyelitis Optica; Multiple Sclerosis; Myasthenia Gravis; Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Skin Diseases

Study Officials

• Nan Shen, MD & PhD

  Department of Rheumatology, RenJi Hospital, School of Medicine, Shanghai JiaoTong University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nan Shen, MD & PhD

CONTACT

+86-21-63260477; nanshensibs@gmail.com

Huihua Ding, MD

CONTACT

+86-21-53882280; dinghuihua@outlook.com

Study Design

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

Study Record Dates

• First Submitted: January 21, 2024
• First Posted: February 8, 2024
• Study Start: March 20, 2024
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2040
• Last Updated: July 25, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)