A Study of LCAR-AIO in Subjects With Relapsed/Refractory Autoimmune Diseases

July 2, 2025 updated by: Nanjing Legend Biotech Co.

An Open-label Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of LCAR-AIO for the Treatment of Relapsed/Refractory Autoimmune Diseases (r/r AID)

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a prospective, single-arm, open-label exploratory clinical study to evaluate the safety, tolerability, pharmacokinetics and efficacy profiles of LCAR-AIO, a chimeric antigen receptor (CAR) -T cell therapy in subjects with relapsed/refractory autoimmune diseases. Patients who meet the eligibility criteria will receive LCAR-AIO infusion. The study will include the following sequential stages: screening, apheresis, pre-treatment (cell product preparation: lymphodepleting chemotherapy), treatment (LCAR AIO infusion) and follow-up.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Hefei, China
        • The First Affiliated Hospital of USTC (Anhui Provincial Hospital)
      • Nanjing, China
        • Jiangsu Province Hospital
      • Wenzhou, China
        • The 1st Affiliated Hospital of WHU
      • Xuzhou, China
        • The Affiliated Hospital of Xuzhou Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Subjects voluntarily participate in clinical research.
  2. Age 18-65 years.
  3. Adequate organ function at screening.
  4. Clinical laboratory values meet criteria at screening.

SLE:

  • Have been diagnosed of SLE at least 6 months before screening.
  • At screening, antinuclear antibody, and/or anti-dsDNA antibody, and/or anti-Smith antibody should be positive.
  • Fulfill relapsed/refractory SLE conditions.

AAV:

  • Have been diagnosed of AAV before screening.
  • Positive test for anti-PR3 or anti-MPO antibodies at screening.
  • Fulfill relapsed/refractory AAV conditions.

SSc:

  • Have been diagnosed of SSc before screening.
  • At screening, mRSS is higher than 10.
  • Fulfill relapsed/refractory SSc conditions.

IIM:

  • Have been diagnosed of IIM before screening.
  • Positive test for myositis-associated antibodies or myositis-specific antibodies at screening.
  • Fulfill relapsed/refractory IIM conditions.

Exclusion Criteria:

  1. Active infections such as hepatitis and tuberculosis.
  2. Other autoimmune diseases.
  3. Serious underlying diseases such as tumor, uncontrolled diabetes.
  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.
  5. Participated in other clinical trials within 4 weeks.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Before treatment with LCAR-AIO T cells, subjects will receive a conditioning regimen.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• The SLEDAI-2K scale is a cumulative and weighted index used to assess disease activity across 24 different disease descriptors in patients with SLE. A total score can fall between 0 and 105, with a higher score representing a more significant degree of disease activity.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Change in Birmingham vasculitis activity score (BVAS) from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• The BVAS is a cumulative and weighted index used to assess disease activity across 9 organ systems in patients with AAV. A total score can fall between 0 and 64, with a higher score representing a more significant degree of disease activity.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Change in modified Rodnan skin score (mRSS) from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• The mRSS is a scale used to assess skin thickness and scope of SSc patients by physically evaluating 17 positions. A total score can fall between 0 and 51, with a higher score representing a more significant degree of skin involvement.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Change in manual muscle testing (MMT-8) score from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• This validated tool assesses muscle strength through muscle testing an abbreviated group of 8 proximal, distal, and axial muscles in IIM patients. A total score can fall between 0 and 150, with a lower score representing a more significant degree of muscle involvement.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Change in Physician global assessment (PGA) scores from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• The physician will evaluating the global activity of AID patients by placing a vertical line on a 100-mm visual analog scale on which the left-hand boundary represents "no activity", and the right-hand boundary represents "the most severe activity".
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Changes from baseline in immunological markers and Immunogenicity (anti-drug antibody) from baseline up to 104 weeks
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• Detection of Immunoglobulins, autoantibodies (anti-dsDNA antibody, anti-Smith antibody, etc.) and anti-drug antibody.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence, severity, and type of treatment-emergent adverse events (TEAEs)
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• 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.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Incidence of dose-limiting toxicity (DLT)
Time Frame: 30 days after LCAR-AIO infusion (Day 1)
• 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)
Pharmacokinetics in peripheral blood
Time Frame: Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
• CAR positive T cells levels in peripheral blood after LCAR-AIO infusion.
Minimum 104 Weeks after LCAR-AIO infusion (Day 1)
Recommended Dose regimen finding
Time Frame: Maximum 104 Weeks after LCAR-AIO infusion (Day 1)
Recommended Dose regimen established through dose exploratory.
Maximum 104 Weeks after LCAR-AIO infusion (Day 1)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Nanjing Legend Biotech Co.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 17, 2025

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 17, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 17, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 4, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 4, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 10, 2025

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 8, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 2, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • LB2305-0002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings