Actively Recruiting

Phase 1
Age: 18Years - 70Years
All Genders
ID06869278

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

Led by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology · Updated on 2025-06-11

37

Participants Needed

3

Research Sites

182 weeks

Total Duration

On this page

Sponsors

U

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Lead Sponsor

N

Nanjing Legend Biotech Co.

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are investigating LCAR-AIO, a type of CAR-T cell therapy, for adults with relapsed or refractory neurological autoimmune diseases including Multiple Sclerosis (MS), Neuromyelitis Optica Spectrum Disease (NMOSD), Anti-Myelin Oligodendrocyte Glycoprotein-IgG Associated Disorders (MOGAD), and Myasthenia Gravis (MG). This phase 1, open-label, single-arm study aims to explore the safety, tolerability, pharmacokinetics, and effectiveness of LCAR-AIO in these patients who have not responded well to prior treatments. Participants will receive a single infusion of LCAR-AIO CAR-T cells after a preparation process involving screening, apheresis to collect cells, and pre-treatment with lymphodepleting chemotherapy. The study includes sequential stages: screening, cell collection, cell preparation, treatment infusion, and follow-up monitoring. The infusion is given once per participant at prescribed dose levels. Throughout the study, participants will undergo assessments including monitoring for adverse events and dose-limiting toxicities, measuring CAR-T cell concentrations and gene expression levels, and evaluating clinical outcomes such as relapse rates, disability scores, visual acuity, pain levels, and functional scales related to their conditions. These evaluations will be conducted from baseline through up to 104 weeks post-infusion, allowing long-term safety and efficacy tracking. The study duration allows detailed observation and follow-up of participants after treatment.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 70Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Voluntarily agree to participate in clinical research
  • Age between 18 and 70 years
  • Adequate organ function at screening
  • Clinical laboratory values meet criteria at screening visit
  • Diagnosed with Multiple Sclerosis at least 6 months before screening and have relapsed/refractory MS
  • Diagnosed with NMOSD or MOGAD at least 6 months before screening, with positive AQP-4 IgG (NMOSD) or MOG-IgG (MOGAD) by cell-based assay, and have relapsed/refractory condition
  • Diagnosed with Myasthenia Gravis at least 6 months before screening, with positive AChR-IgG or MuSK-IgG, and have relapsed/refractory condition
Not Eligible

You will not qualify if you...

  • Active infections such as hepatitis or tuberculosis
  • Other autoimmune diseases besides the study conditions
  • Serious underlying diseases such as tumor, uncontrolled diabetes, or significant cardiovascular disease
  • Female participants who are pregnant, breastfeeding, or planning pregnancy during the study or within 1 year after treatment

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Apheresis

Duration - 1 to 2 days

Participants undergo apheresis to collect cells for CAR-T cell preparation.

1 visit (in-person)

Pre-treatment

Duration - Several days

Participants receive lymphodepleting chemotherapy as preparation before the CAR-T cell infusion.

Daily visits during chemotherapy days

Treatment

Duration - 1 day

Participants receive a single infusion of LCAR-AIO CAR-T cells as the active treatment for their neurological autoimmune disease.

1 infusion visit (in-person)

Follow-up

Duration - Up to 104 weeks

Participants are monitored up to 104 weeks after infusion to evaluate safety, efficacy, and long-term outcomes.

Regular follow-up visits during 2 years post-treatment

Trial Site Locations

Total: 3 locations

1

The Affiliated Hospital of Fujian Medical University

Fuzhou, China

Not Yet Recruiting

2

The First Affiliated Hospital of SOOCHOW University

Suzhou, China

Actively Recruiting

3

Union Hospital Tongji Medical College HUAZHONG University of Science and Technology

Wuhan, China

Actively Recruiting

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Research Team

Q

Qiubai li

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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