A Study of LCAR-AIO CAR-T Cells for Treating Relapsed/Refractory Neurological Autoimmune Diseases

NCT06869278 | Recruiting Phase 1

• 1 other identifier: LB2305-0003
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 3

Brief Summary

This is a prospective, single-arm, open-label, dose-exploration and expansion clinical study of LCAR-AIO in adult subjects with relapsed/refractory neurological autoimmune diseases.

Conditions

Multiple Sclerosis (MS); Neuromyelitis Optica Spectrum Disease (NMOSD); Anti-Myelin Oligodendrocyte Glycoprotein-IgG Associated Disorders (MOGAD); Myasthenia Gravis

Keywords

Relapsed/Refractory Multiple Sclerosis (r/r MS); Relapsed/Refractory Neuromyelitis Optica Spectrum Disease (r/r NMOSD); Relapsed/Refractory Anti-Myelin Oligodendrocyte Glycoprotein-IgG Associated Disorders (r/r MOGAD); Relapsed/Refractory Myasthenia Gravis (r/r MG)

Outcome Measures

Primary Outcomes (5)

• Incidence, severity, and type of treatment-emergent adverse events (TEAEs)

  An adverse event refers to any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product (investigational or non-investigational), which does not necessarily have a causal relationship with the treatment.

  Baseline to 104 Weeks after last subject infusion

• Incidence of dose-limiting toxicity (DLT)

  DLTs are severe adverse events refers to any untoward medical occurred in a subject participated in a clinical investigation, which have a causal relationship with the treatment and will limit the dose escalation.

  30 days after LCAR-AIO infusion (Day 1)

• Chimeric Antigen Receptor T (CAR-T) Positive Cell Concentration

  Venous blood samples will be collected for measurement of CAR-T positive cellular concentration.

  Baseline to 104 Weeks after last subject infusion

• Recommended Phase 2 Dose (RP2D) regimen finding

  RP2D established through dose exploratory.

  Baseline to 104 Weeks after last subject infusion

• Transgene Levels of LCAR-AIO CAR-T Cells

  Transgene Levels of LCAR-AIO CAR-T Cells using sensitive assay methods will be assessed

  Baseline to 104 Weeks after last subject infusion

Secondary Outcomes (7)

• Annua Relapse Rate (ARR)

  Baseline to 104 Weeks after last subject infusion

• Change in Expanded Disability Status Scale (EDSS) scores from baseline up to 104 weeks

  Baseline to 104 Weeks after last subject infusion

• Changes in Visual Acuity from baseline up to 104 weeks

  Baseline to 104 Weeks after last subject infusion

• Changes in Visual analogue scale (VAS) pain score from baseline up to 104 weeks

  Baseline to 104 Weeks after last subject infusion

• Changes in MSE proportion from baseline up to 104 weeks

  Baseline to 104 Weeks after last subject infusion

Study Arms (1)

LCAR-AIO T Cells

EXPERIMENTAL

Chimeric antigen receptor T cells (LCAR-AIO) Each subject will be given a single-dose LCAR-AIO cells infusion at each dose level

Biological: LCAR-AIO T cells

Interventions

LCAR-AIO T cells BIOLOGICAL

Before treatment with LCAR-AIO T cells, subjects will receive a conditioning regimen.

LCAR-AIO T Cells

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects voluntarily participate in clinical research.
• Age 18-70 years.
• Adequate organ function at screening.
• Clinical laboratory values meet criteria at screening visit.
• Indications include:
• MS;
• Have been diagnosed of MS at least 6 months before screening.
• Fulfill relapsed/refractory MS conditions.
• NMOSD/MOGAD:
• Have been diagnosed of NMOSD/MOGAD at least 6 months before screening.
• AQP-4 IgG (NMOSD), or MOG-IgG (MOGAD) should be positive by CBA (Cell based transfection immunofluorescence assay).
• Fulfill relapsed/refractory NMOSD/MOGAD conditions.
• MG:
• Have been diagnosed of MG at least 6 months before screening.
• AChR-IgG or MuSK-IgG should be positive.

You may not qualify if:

• Active infections such as hepatitis and tuberculosis.
• Other autoimmune diseases.
• Serious underlying diseases such as tumor, uncontrolled diabetes and clinically significant cardiovascular disease.
• 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.

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead
• Nanjing Legend Biotech Co.: collaborator

Study Sites (3)

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

MeSH Terms

Conditions

Multiple Sclerosis; Myasthenia Gravis; Recurrence

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Qiubai li

CONTACT

18221636229; Tingyu.Zhang@legendbiotech.cn

Study Design

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

Study Record Dates

• First Submitted: February 25, 2025
• First Posted: March 11, 2025
• Study Start: June 17, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2029
• Last Updated: June 11, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)