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

Fudan University

Status and phase: Not yet enrolling; Phase 1

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

Treatments: Biological: CD20/BCMA-directed CAR-T cells

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT07341828

KY2025-1015

Details and patient eligibility

About

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

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 18 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.

Exclusion criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 1 patient group

C-CAR168

Experimental group

Description:

Autologous C-CAR168 administered by intravenous (IV) infusion

Treatment:

Biological: CD20/BCMA-directed CAR-T cells