LCAR-AIO CAR-T treatment for relapsed or treatment-resistant neurological autoimmune diseases

An Open Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCAR-AIO CAR-T Cells for Treating Relapsed/Refractory Neurological Autoimmune Diseases

Doctors will try a CAR-T cell therapy called LCAR-AIO in adults whose multiple sclerosis, NMOSD, MOGAD, or myasthenia gravis has not responded to other treatments.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 37 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology (Academic / other)
• Drugs / interventions: chimeric antigen receptor, chemotherapy
• Locations: 3 sites (Fuzhou and 2 other locations)
• Trial ID: NCT06869278 on ClinicalTrials.gov

What this trial studies

This phase 1, single-arm, open-label study uses dose exploration followed by an expansion cohort to test safety, tolerability, pharmacokinetics, and early signs of efficacy of LCAR-AIO CAR-T cells in adults with relapsed or refractory neurological autoimmune diseases. Eligible participants undergo screening and apheresis to collect cells, receive lymphodepleting chemotherapy while their CAR-T product is manufactured, then receive a single infusion of LCAR-AIO and scheduled follow-up visits. The study will monitor adverse events, CAR-T cell persistence and distribution, and clinical measures of neurologic disease activity. Results from dose escalation will guide the expansion cohort to gather additional safety and preliminary efficacy data.

Who should consider this trial

Good fit: Adults 18–70 with relapsed or refractory MS, AQP4+ NMOSD, MOG‑IgG+ MOGAD, or antibody‑positive myasthenia gravis who meet organ function and laboratory criteria and can undergo apheresis and lymphodepleting chemotherapy.

Not a fit: Patients with active infections, other significant autoimmune diseases, major uncontrolled comorbidities, or those lacking the required disease-specific antibodies are unlikely to benefit or to qualify.

Why it matters

Potential benefit: If successful, the therapy could produce durable disease control or remission for patients who have failed existing treatments.

How similar studies have performed: Early case reports and small studies of CAR-T therapies in refractory autoimmune diseases have shown promising remissions, but CAR-T for neurological autoimmune disorders remains experimental.

Eligibility criteria

Inclusion Criteria:

1. Subjects voluntarily participate in clinical research.
2. Age 18-70 years.
3. Adequate organ function at screening.
4. Clinical laboratory values meet criteria at screening visit.
5. Indications include:

MS;

1. Have been diagnosed of MS at least 6 months before screening.
2. Fulfill relapsed/refractory MS conditions.

NMOSD/MOGAD:

1. Have been diagnosed of NMOSD/MOGAD at least 6 months before screening.
2. AQP-4 IgG (NMOSD), or MOG-IgG (MOGAD) should be positive by CBA (Cell based transfection immunofluorescence assay).
3. Fulfill relapsed/refractory NMOSD/MOGAD conditions.

MG:

1. Have been diagnosed of MG at least 6 months before screening.
2. AChR-IgG or MuSK-IgG should be positive.
3. Fulfill relapsed/refractory NMOSD/MOGAD conditions.

Exclusion Criteria:

1. Active infections such as hepatitis and tuberculosis.
2. Other autoimmune diseases.
3. Serious underlying diseases such as tumor, uncontrolled diabetes and clinically significant cardiovascular disease.
4. Female subjects who were pregnant, breastfeeding, or planning to become pregnant while participating in this study or within 1 year of receiving LCAR-AIO treatment.

Where this trial is running

Fuzhou and 2 other locations

• The Affiliated Hospital of Fujian Medical University — Fuzhou, China (Not_yet_recruiting)
• The First Affiliated Hospital of SOOCHOW University — Suzhou, China (Recruiting)
• Union Hospital Tongji Medical College HUAZHONG University of Science and Technology — Wuhan, China (Recruiting)

Study contacts

• Study coordinator: Qiubai li

Conditions Multiple Sclerosis; Neuromyelitis Optica Spectrum Disease; Anti-Myelin Oligodendrocyte Glycoprotein-IgG Associated Disorders; Myasthenia Gravis; Relapsed/Refractory Multiple Sclerosis; Relapsed/Refractory Neuromyelitis Optica Spectrum Disease; Relapsed/Refractory Anti-Myelin Oligodendrocyte Glycoprotein-IgG Associated Disorders; Relapsed/Refractory Myasthenia Gravis