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

NCT07341828 | Not Yet Recruiting Phase 1

• 1 other identifier: KY2025-1015
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Stiff Person Syndrome; Autoimmune Encephalitis; Multiple Sclerosis (MS); Neuromyelitis Optica Spectrum Disorders (NMOSD)

Keywords

CD20/BCMA-directed CAR-T cells

Outcome Measures

Primary Outcomes (2)

• Incidence and severity of Adverse Events [Safety and Tolerability]

  Incidence and severity of adverse events (AE) and serious adverse events (SAE) within three months following infusion

  Throughout the first 3 months follow up period completion

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

  Based on the assessment of overall safety profile

  Throughout the first 24 months follow up period completion

Secondary Outcomes (14)

• Incidence and severity of adverse events (AE)

  Throughout the first 24 months follow up period completion

• MS: No Evidence of Disease Activity-3 (NEDA-3)

  Throughout the first 24 months follow up period completion

• MS and NMOSD: Expanded Disability Status Scale (EDSS)

  Throughout the first 24 months follow up period completion

• MS and NMOSD: MRI

  Throughout the first 24 months follow up period completion

• Autoimmune Encephalitis (AiE): Clinical Assessment Scale in Autoimmune Encephalitis (CASE)

  Throughout the first 24 months follow up period completion

Other Outcomes (3)

• Serum cytokines changes

  Throughout the first 24 months follow up period completion

• Soluble BCMA changes in peripheral blood

  Throughout the first 24 months follow up period completion

• Changes in CSF CAR DNA copy number and CAR-T cells

  Throughout the first 24 months follow up period completion

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 Multiple Sclerosis (MS)/Neuromyelitis Optica Spectrum Disorders (NMOSD)/Autoimmune Encephalitis(AiE)/Stiff Person Spectrum Disorder(SPSD) according to recognized diagnostic criteria for at least 6 months.
• Prior treatment failure with standard therapy.
• 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.
• A history of ≥ Grade 2 bleeding within 4 weeks 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.
• Any contraindication to lumbar puncture.

Sponsors & Collaborators

• Huashan Hospital: lead
• Shanghai AbelZeta Ltd.: collaborator

MeSH Terms

Conditions

Multiple Sclerosis; Neuromyelitis Optica; Autoimmune Diseases of the Nervous System; Stiff-Person Syndrome

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases

Central Study Contacts

Xiangjun Chen

CONTACT

+86 21 52888159; xiangjchen@fudan.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: January 6, 2026
• First Posted: January 14, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): February 1, 2029
• Last Updated: January 15, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share