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

NCT06866080 | Withdrawn Early Phase 1

• 1 other identifier: LB2305-0002
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

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 autoimmune diseases.

Conditions

Relapsed/Refractory Autoimmune Diseases

Keywords

Relapsed/Refractory Systemic lupus erythematosus (r/r SLE); Relapsed/Refractory Systemic Sclerosis (SSc); ), Relapsed/Refractory Anti-neutrophil cytoplastic antibodies (ANCA)-associated vasculitis (AAV); Idiopathic Relapsed/Refractory Inflammatory Myopathies (IIM)

Outcome Measures

Primary Outcomes (6)

• Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline up to 104 weeks

  • 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

  • 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

  • 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

  • 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

  • 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

  • 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 Outcomes (4)

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

  Minimum 104 Weeks after LCAR-AIO infusion (Day 1)

• Incidence of dose-limiting toxicity (DLT)

  30 days after LCAR-AIO infusion (Day 1)

• Pharmacokinetics in peripheral blood

  Minimum 104 Weeks after LCAR-AIO infusion (Day 1)

• Recommended Dose regimen finding

  Maximum 104 Weeks after LCAR-AIO infusion (Day 1)

Study Arms (1)

Chimeric antigen receptor T cells (LCAR-AIO)

EXPERIMENTAL

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.

Chimeric antigen receptor T cells (LCAR-AIO)

Eligibility Criteria

• Age: 18 Years - 65 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 18-65 years.
• Adequate organ function at screening.
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Nanjing Legend Biotech Co.: lead

Study Sites (4)

The First Affiliated Hospital of USTC (anhui provincial hospital)

Hefei, China

Location

Jiangsu Province Hospital

Nanjing, China

Location

The 1st Affiliated Hospital of WHU

Wenzhou, China

Location

The Affiliated Hospital of XUZHOU Medical University

Xuzhou, China

Location

MeSH Terms

Conditions

Autoimmune Diseases; Recurrence; Lupus Erythematosus, Systemic; Scleroderma, Systemic; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Myositis

Condition Hierarchy (Ancestors)

Immune System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2025
• First Posted: March 10, 2025
• Study Start: June 17, 2025
• Primary Completion: June 17, 2025
• Study Completion: June 17, 2025
• Last Updated: July 8, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (4)