Treatment for autoimmune diseases using LCAR-AIO therapy

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 study is testing a new treatment called LCAR-AIO therapy to see if it can help adults with tough autoimmune diseases feel better.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 38 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Nanjing Legend Biotech Co. (Industry-sponsored)
• Drugs / interventions: chimeric antigen receptor, chemotherapy
• Locations: 4 sites (Hefei and 3 other locations)
• Trial ID: NCT06866080 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of LCAR-AIO, a chimeric antigen receptor T cell therapy, in adults with relapsed or refractory autoimmune diseases. Participants will undergo a series of procedures including screening, apheresis, lymphodepleting chemotherapy, and LCAR-AIO infusion. The study aims to explore the pharmacokinetics and tolerability of the treatment in conditions such as systemic lupus erythematosus, systemic sclerosis, ANCA-associated vasculitis, and inflammatory myopathies. It is designed as a single-arm, open-label trial to gather preliminary data on the therapy's effectiveness.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with relapsed or refractory autoimmune diseases such as systemic lupus erythematosus, systemic sclerosis, ANCA-associated vasculitis, or inflammatory myopathies.

Not a fit: Patients with active infections or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat autoimmune diseases.

How similar studies have performed: While CAR-T cell therapies have shown promise in oncology, their application in autoimmune diseases is still novel and largely untested.

Eligibility criteria

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.

Where this trial is running

Hefei and 3 other locations

• The First Affiliated Hospital of USTC (anhui provincial hospital) — Hefei, China (Recruiting)
• Jiangsu Province Hospital — Nanjing, China (Recruiting)
• The 1st Affiliated Hospital of WHU — Wenzhou, China (Not_yet_recruiting)
• The Affiliated Hospital of XUZHOU Medical University — Xuzhou, China (Recruiting)

Conditions Relapsed/Refractory Autoimmune Diseases; Relapsed/Refractory Systemic lupus erythematosus; Relapsed/Refractory Systemic Sclerosis; ), Relapsed/Refractory Anti-neutrophil cytoplastic antibodies-associated vasculitis; Idiopathic Relapsed/Refractory Inflammatory Myopathies