Clinical trial NCT06939166: Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases
Official title: A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapsed / Refractory Neurological Autoimmune Diseases
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
Conditions: Neuromyelitis Optica Spectrum Disorders
Myasthenia Gravis
Multiple Sclerosis
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Autoimmune Encephalitis
Keywords: Universal Allogeneic CAR T-cells
Lead sponsor: Tianjin Huanhu Hospital
Sponsor class: OTHER
Collaborators: name: Shanghai Xiniao Biotech Co., Ltd.
First posted: 2025-04-22
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.

Detailed description: This is an investigator-initiated trial to evaluate the safety and efficacy of universal CD19/BCMA CAR T-cells in Relapsed or Refractory Neurological Autoimmune Diseases.

Study intervention consists of a single infusion of universal CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.

Interim analysis will be performed when participants finish the visit 90 days after CAR T-cell infusion.

Eligibility criteria: Inclusion Criteria:

* Age ≥ 18 years.
* Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
* Subjects with relapsed or refractory neurological autoimmune diseases, Including neuromyelitis optica spectrum disorders(NMOSD), myasthenia gravis(MG), multiple sclerosis(MS)，Autoimmune encephalitis(AE) and chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP).
* Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
* Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.

Exclusion Criteria:

* Subjects with a history of severe drug allergies or allergic tendencies.
* History of malignancy within five years.
* Subjects with insufficient cardiac function.
* Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA \>the upper limit of detection; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing.
* Pregnant women or women planning to conceive.

Primary outcomes:
measure: The number and severity of dose-limiting toxicity (DLT) events
timeFrame: Within 28 Days After UCAR T-cell Infusion
measure: The total number, incidence, and severity of AEs
timeFrame: Up to 90 days After UCAR T-cell Infusion
Secondary outcomes:
measure: NMOSD、MS: Expanded Disability Status Scale (EDSS) score
timeFrame: Up to 24 Months After UCAR T-cell Infusion
measure: NMOSD、MS: Modified Rankin Scale
timeFrame: Up to 24 Months After UCAR T-cell Infusion
measure: MG: Quantitative Myasthenia Gravis Score (QMG)
timeFrame: Up to 24 Months After UCAR T-cell Infusion
measure: MG: Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
timeFrame: Up to 24 Months After UCAR T-cell Infusion
measure: CIDP: Inflammatory Neuropathy Cause and Treatment (INCAT) Score
timeFrame: Up to 24 Months After UCAR T-cell Infusion
measure: AE: Change in CASE
timeFrame: Up to 24 Months After UCAR T-cell Infusion
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Tianjin Huanhu Hospital
Study officials:
References: