A Clinical Study of TI-0032-III Injection in Patients With Relapsed and Refractory Autoimmune Diseases

NCT07413341 | Recruiting Phase 1

• 1 other identifier: 0032-III-101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label, dose escalation study in patients with relapsed and refractory autoimmune diseases. Study drug, TI-0032-III injection, is composed of lipid nanoparticles (LNPs) targeting T cells that encapsulate circular RNA encoding the CD19 chimeric antigen receptor (CAR), which is a therapeutic biological product. It is clinically intended for the treatment of various relapsed and refractory B cell-related autoimmune diseases, such as systemic lupus erythematosus, sjögren's syndrome, systemic sclerosis, idiopathic inflammatory myositis, and antiphospholipid syndrome.

Conditions

Systemic Lupus Erythematosus (SLE); Sjogren's Syndrome (SS); Systemic Sclerosis (SSc); Inflammatory Myopathy; ANCA Associated Systemic Vasculitis

Outcome Measures

Primary Outcomes (1)

• Safety outcomes

  Safety assessments are conducted using the NCI-CTCAE version 5.0 standards

  From first dose of TI-0032-III injection until 12 months

Secondary Outcomes (5)

• Efficacy outcome for systemic lupus erythematosus

  From first dose of TI-0032-III injection until 3 months

• Efficacy outcome for Sjögren's syndrome

  From first dose of TI-0032-III injection until 3 months

• Efficacy outcome for systemic sclerosis

  From first dose of TI-0032-III injection until 3 months

• Efficacy outcome for inflammatory myopathy

  From first dose of TI-0032-III injection until 3 months

• Efficacy outcome for ANCA-associated vasculitis

  From first dose of TI-0032-III injection until 3 months

Other Outcomes (4)

• Exploratory PK outcomes

  From first dose of TI-0032-III injection until 3 months

• Exploratory PK outcomes

  From first dose of TI-0032-III injection until 3 months

• Exploratory PD outcome

  From first dose of TI-0032-III injection until 12 months

Study Arms (1)

TI-0032-III injection

EXPERIMENTAL

In vivo CD19-targeted Chimeric Antigen Receptor (CAR) T Cell Therapy

Biological: TI-0032-III injection

Interventions

TI-0032-III injection BIOLOGICAL

Multiple doses of TI-0032-III injection will be infused

TI-0032-III injection

Eligibility Criteria

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

You may qualify if:

• Understand trial procedures and methods, voluntarily sign the informed consent form .
• Age ranges from 18 to 65 years old (including threshold), regardless of gender.
• Positive expression of CD19 on peripheral blood B cells determined by flow cytometry.
• Bone marrow function: neutrophil count ≥ 1.5 × 10\^9/L, lymphocyte count ≥ 0.8 × 10\^9/L, hemoglobin ≥ 90 g/L, platelet ≥ 100 × 10\^9/L. Blood transfusion and growth factors must not be used within 14 days prior to screening to meet the above requirements.
• Coagulation function: international normalized ratio or activated partial thromboplastin time ≤ 1.5× upper limit of normal range (ULN).
• Cardiopulmonary function: left ventricular ejection fraction ≥ 50% on echocardiography; for lung function, dyspnea ≤Grade 1 of the NCI-CTCAE version 5.0 standards when breathing room air, and pulse oximetry ≥ 92%.
• Liver function: alanine aminotransferase ≤ 1.5 × ULN, aspartate aminotransferase ≤ 1.5 × ULN, total bilirubin ≤ 1.5 × ULN (total bilirubin at least ≤ 3.0 mg/dL in patients with Gilbert syndrome).
• Renal function: creatinine clearance (by Cockcroft-Gault formula) ≥ 50 mL/min.
• Criteria for SLE：
• Meet the 2019 European Alliance of Associations for Rheumatology/American College of Rheumatology (EULAR/ACR) classification criteria for SLE;
• SLEDAI-2000 score \> 6 in the moderate to severe active phase of the disease;
• And have at least one British Isles Lupus Assessment Group (BILAG-2004) grade A (severe manifestation) or two grade B (moderate manifestation) organ scores, or both.
• Ineffective conventional treatment or relapse of disease activity after remission. Definition of routine treatment: Use of glucocorticoids (Above 1 mg/kg/d) and cyclophosphamide, and any of the following immunomodulatory drugs for more than 6 months: antimalarials, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, and biologics such as rituximab, belimumab, and telitacicept.
• Criteria for Sjögren's syndrome:
• Meet the 2002 AECG criteria for primary Sjögren's syndrome or the 2016 ACR/EULAR classification criteria;

You may not qualify if:

• Prohibited medications and treatments:
• Any RNA-LNP products or other LNP drugs received within the past two years.
• Received intravenous gamma immunoglobulin within 24 weeks prior to screening.
• Received plasmapheresis therapy within 12 weeks prior to screening.
• Subjects who have not fully recovered from surgery within 4 weeks prior to screening.
• Epilepsy or use of psychotropic or sedative drugs during the screening period (except medications for sleeping).
• Received any live or live attenuated vaccine within 12 weeks prior to screening, or requiring vaccination at any time during clinical trial treatment.
• Participated in clinical trial of other drugs or medical devices within 12 weeks prior to screening (except for those whose screening failed).
• Concomitant diseases or clinical condition:
• History of lupus nephropathy requiring hemodialysis or treatment with high-dose corticosteroids (\> 100 mg/d prednisone or equivalent) within 90 days prior to baseline.
• Epilepsy or use of psychotropic or sedative drugs during the screening period (except medications for sleeping).
• Received any live or live attenuated vaccine within 12 weeks prior to screening, or requiring vaccination at any time during clinical trial treatment.
• Participated in clinical trial of other drugs or medical devices within 12 weeks prior to screening (except for screen failures).
• Have active central nervous system lupus, including meningitis aseptic, cerebral vasculitis, demyelinating syndrome, myelopathy, acute demented, psychosis, acute inflammatory demyelinating polyradiculoneuropathy, mononeuropathy (single/multiple), cranial neuropathy, plexopathy, status epilepticus or cerebellar ataxia.
• Presence of other systemic inflammations such as, but not limited to, rheumatoid arthritis, juvenile chronic arthritis, spondyloarthritis, Crohn's disease, ulcerative colitis, or psoriatic arthritis within 12 weeks prior to screening (except for those with secondary Sjögren's syndrome, who should not be excluded).

Sponsors & Collaborators

• Therorna: lead

Study Sites (1)

Shanghai Changzheng Hospital

Shanghai, Shanghai Municipality, 200003, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Sjogren's Syndrome; Scleroderma, Systemic; Myositis

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Dry Eye Syndromes; Lacrimal Apparatus Diseases; Eye Diseases; Skin Diseases; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Hu ji Xu

  Shanghai Changzheng Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiao bing Wang, PhD

CONTACT

021-81886999; gale820907@163.com

Hu ji Xu, PhD

CONTACT

Study Design

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

Study Record Dates

• First Submitted: February 2, 2026
• First Posted: February 17, 2026
• Study Start: January 22, 2026
• Primary Completion: May 1, 2026
• Study Completion (Estimated): January 30, 2027
• Last Updated: February 17, 2026

Record last verified: 2026-02

Locations

CN (1)