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NCT Number: NCT07341828

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

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

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Key information

Conditions

Multiple Sclerosis (MS) Autoimmune Diseases Autoimmune Diseases of the Nervous System Autoimmune Encephalitis Central Nervous System Diseases Cranial Nerve Diseases Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Eye Diseases Immune System Diseases Multiple Sclerosis Myelitis, Transverse Nervous System Diseases Neuromuscular Diseases Neuromyelitis Optica Neuromyelitis Optica Spectrum Disorders (NMOSD) Optic Nerve Diseases Optic Neuritis Spinal Cord Diseases Stiff Person Syndrome Stiff-Person Syndrome

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CD20/BCMA-directed CAR-T cells

Biological

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

Other names: C-CAR168

Primary outcomes

  1. Incidence and severity of Adverse Events [Safety and Tolerability]

    Time frame: Throughout the first 3 months follow up period completion

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

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

    Time frame: Throughout the first 24 months follow up period completion

    Based on the assessment of overall safety profile

Secondary outcomes

  1. Incidence and severity of adverse events (AE)

    Time frame: Throughout the first 24 months follow up period completion

    Incidence and severity of adverse events (AE) and serious adverse events (SAE) during the study

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

    Time frame: Throughout the first 24 months follow up period completion

    Proportion of participants achieving NEDA-3 at 6 months post-infusion and during the study period

  3. MS and NMOSD: Expanded Disability Status Scale (EDSS)

    Time frame: Throughout the first 24 months follow up period completion

    Change from baseline in EDSS at 6 months and 24 months post-infusion. EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death).

  4. MS and NMOSD: MRI

    Time frame: Throughout the first 24 months follow up period completion

    Number of T1 gadolinium-enhancing lesions and new or enlarging T2 lesions, as well as their change from baseline, at 6 months and 24 months post-infusion. Change from baseline in total T2 lesion volume, gray matter volume (GMV), white matter volume (WMV), and brain volume (BV), as well as annualized-brain volume loss (a-BVL), at 6 months and 24 months post-infusion

  5. Autoimmune Encephalitis (AiE): Clinical Assessment Scale in Autoimmune Encephalitis (CASE)

    Time frame: Throughout the first 24 months follow up period completion

    Change from baseline in CASE at 6 months and 24 months post-infusion. The Clinical Assessment Scale in Autoimmune Encephalitis (CASE) has a score range from 0 to 27, and higher scores indicate a worse clinical outcome.

  6. MS, NMOSD and AiE: Annualized Relapse Rate (ARR)

    Time frame: Throughout the first 24 months follow up period completion

    ARR at 6 months and 24 months post-infusion

  7. Stiff-Person Syndrome (SPS): Distribution of Stiffness Index (DSI)

    Time frame: Throughout the first 24 months follow up period completion

    Change from baseline in DSI at 6 months and 24 months post-infusion. Distribution of Stiffness Index (DSI) is a validated indicator or stiffness. Scores range from 0 to 6 and reflect the extent of stiffness. Higher scores indicate a worse outcome.

  8. SPS: Heightened Sensitivity Score (HSS)

    Time frame: Throughout the first 24 months follow up period completion

    Change from baseline in HSS at 6 months and 24 months post-infusion. Heightened Sensitivity Score (HSS) measures changes in the frequency of spasms. Scores range from 0 to 7. Higher scores indicate a worse outcome.

  9. Pharmacokinetics (PK): Maximal plasma concentration (Cmax)

    Time frame: Throughout the first 24 months follow up period completion

    Cmax of C-CAR168 in peripheral blood

  10. PK: Time to reach the maximal plasma concentration (Tmax)

    Time frame: Throughout the first 24 months follow up period completion

    Tmax of C-CAR168 in peripheral blood

  11. PK: Duration in peripheral blood (Tlast)

    Time frame: Throughout the first 24 months follow up period completion

    Tlast of C-CAR168 in peripheral blood

  12. PK: Area under curve (AUC)

    Time frame: Throughout the first 24 months follow up period completion

    AUC of C-CAR168 in peripheral blood

  13. Pharmacodynamics (PD): Depletion of peripheral blood B cells, plasma cells, and CD20dim T cells

    Time frame: Throughout the first 24 months follow up period completion

  14. PD: Decline of serum immunoglobulin

    Time frame: Throughout the first 24 months follow up period completion

Other outcomes

  1. Serum cytokines changes

    Time frame: Throughout the first 24 months follow up period completion

    Detection of serum cytokines changes over time by flow cytometry

  2. Soluble BCMA changes in peripheral blood

    Time frame: Throughout the first 24 months follow up period completion

    Detection of soluble BCMA changes in peripheral blood by Enzyme Linked ImmunoSorbent Assay (ELISA)

  3. Changes in CSF CAR DNA copy number and CAR-T cells

    Time frame: Throughout the first 24 months follow up period completion

    Detection of changes in CSF CAR DNA copy number and CAR-T cells by quantitative polymerase chain reaction (qPCR) and flow cytometry

Study contacts

Contact information is provided by the study sponsor or research team.

Xiangjun Chen

CONTACT

[email protected]

+86 21 52888159

Sponsors and collaborators

Lead sponsor

Huashan Hospital

Other

Collaborators

  • Shanghai AbelZeta Ltd.

Registry information

Official study title

An Exploratory Clinical Study of Anti-CD20/B-cell Maturation Antigen(BCMA) Chimeric Antigen Receptor Autologous T Cell Product (C-CAR168) in the Treatment of Central Nervous System Autoimmune Diseases Refractory to Standard Therapy

Important dates

Study start
2026
Primary completion
2027
Study completion
2029
First posted
Jan 14, 2026
Registry last updated
Jan 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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