Evaluation of Safety, Pharmacokinetics and Pharmacodynamics of Arnovie101, an mRNA-LNP-Based In Vivo CAR-T Therapy, for the Treatment of B Cell-Mediated Autoimmune Diseases (SLE and AIHA)

NCT07629596 | Recruiting Early Phase 1 DMC

• 1 other identifier: BOJI2026007
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open lable and single arm study designed to evaluate the safety, PK and PD of Arnovie101 in B cell-mediated Autoimmune Disease

Conditions

Autoimmune Hemolytic Anemia (AIHA); Systemic Lupus Erythematosus

Keywords

SLE; AIHA; in vivo CAR-T; mRNA-LNP; autoimmune diseases

Outcome Measures

Primary Outcomes (2)

• Incidence of dose-limiting toxicity (DLT)

  Up to 12 months

• Incidence and severity of adverse event (AE) and serious adverse event (SAE)

  Up to 12 months

Secondary Outcomes (13)

• Change from baseline of SLEDAI-2K score after Arnovie101 administration in participants with relapsed/refractory SLE.

  Up to 12 months

• Quantify the clinical activity of Arnovie101 in patients using Physician Global Assessment (PGA) in participants with relapsed/refractory SLE.

  up to 12 months

• Proportion of participants achieving lupus low disease activity status (LLDAS) in participants with relapsed/refractory SLE.

  up to 12 months

• Proportion of patients achieving DORIS remission after Arnovie101 administration in participants with relapsed/refractory SLE.

  up to 12 months

• Changes in serological markers after Arnovie101 administration in participants with relapsed/refractory SLE.

  up to 12 months

Study Arms (1)

Arnovie101 treatment group

EXPERIMENTAL

Participants will receive Arnovie101 infusion (a nanobody-modified LNP encapsulating mRNA for in vivo CAR-T therapy) at the specified dose level and on the specified study days.

Drug: Arnovie101 Infusion Intravenous

Interventions

Arnovie101 Infusion Intravenous DRUG

Dosing will begin at a lower dose level and may be escalated to dose levels considered safe and potentially effective according to the study protocol.

Also known as: in vivo CAR-T

Arnovie101 treatment group

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Ability to voluntarily sign informed consent, including compliance with the requirements and restrictions listed in the Informed Consent Form (ICF) and this protocol.
• Male or female, aged 18 to 60 years (inclusive) at screening.
• Participants with relapsed/refractory SLE with at least 6 months of disease history and meeting the following criteria:
• A. Confirmed diagnosis of SLE according to the 2012 SLICC or 2019 EULAR/ACR revised criteria. SLEDAI-2K score ≥6 at screening. If the score includes low complement and/or anti-dsDNA antibodies, the clinical symptom score of SLEDAI-2K (excluding low complement and/or anti-dsDNA antibodies) should be ≥4. Poor response to standard therapy (at least two first-line treatments, including corticosteroids and immunosuppressants) and disease relapse after treatment.
• B. SLE: Stable standard therapy (including non-steroidal anti-inflammatory drugs, immunosuppressants, biologics, and glucocorticoids; oral glucocorticoid dose of prednisone or equivalent ≥7.5 mg/day and ≤30 mg/day; if combined with an immunosuppressant, no minimum daily dose requirement) for at least 8 weeks before screening, with current dose stable for at least 2 weeks and expected to remain stable during the study. Prior use of at least two immunosuppressants including hydroxychloroquine.
• C. Life expectancy \>6 months.
• Participants with AIHA meeting the following criteria:
• A. Participants with AIHA or Evans syndrome who have failed ≥3 lines of therapy.
• B. Failure of ≥3 lines of therapy must meet all of the following: hemoglobin \<10 g/dL with symptoms of anemia; failure of first-line corticosteroid therapy; failure of second-line rituximab therapy; failure of any one or more third-line regimens (splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, fludarabine, bortezomib, etc.).
• C. Life expectancy \>3 months.
• Adequate organ function:
• A. Renal function: Calculated creatinine clearance (Cockcroft-Gault) ≥30 mL/min without hydration support.
• B. Bone marrow function: Absolute neutrophil count (ANC) ≥1.0×10⁹/L, absolute lymphocyte count (ALC) ≥0.1×10⁹/L, hemoglobin (Hb) ≥60 g/L, platelet count (PLT) ≥20×10⁹/L. Coagulation: International normalized ratio (INR) or activated partial thromboplastin time (APTT) ≤1.5×ULN. Note: No blood transfusion, white blood cell growth factors (e.g., colony-stimulating factors), erythropoietin, or thrombopoietin within 7 days before the laboratory assessment.
• C. Hepatic function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5×ULN, total bilirubin \<2.0 mg/dL (for participants with Gilbert's syndrome, total bilirubin \<3.0 mg/dL; except when caused by SLE itself).
• D. Pulmonary function: Dyspnea ≤CTCAE grade 1 and oxygen saturation (SpO₂) ≥92% on room air (measured by pulse oximeter).

You may not qualify if:

• Presence of an unresected thymoma.
• Pregnant or lactating women.
• History of active severe or unstable neuropsychiatric SLE, including but not limited to poorly controlled seizures, psychosis, acute confusional state, cerebrovascular accident, demyelinating syndrome, cranial neuropathy, or active central nervous system vasculitis.
• Presence of clinically significant central nervous system disease or pathology before screening, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, convulsions/seizures, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis.
• History of major organ transplantation (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/bone marrow transplantation.
• Presence of known active infection at screening, including active tuberculosis, active or infectious pneumonia, or recurrent peptic ulcer; requiring hospitalization, intravenous antibiotics within 4 weeks before screening, or oral antibiotics within 2 weeks before screening; history of herpes zoster within 12 weeks before screening; history of human immunodeficiency virus (HIV) or positive HIV antibody test; positive hepatitis B surface antigen (HBsAg) and/or positive anti-hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA above the lower limit of detection; positive hepatitis C virus (HCV) antibody with detectable HCV RNA above the lower limit of detection; positive syphilis antibody with active infection.
• History of any of the following cardiovascular diseases within 6 months before screening: New York Heart Association (NYHA) class II or higher heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias (e.g., second-degree type II atrioventricular block, third-degree atrioventricular block, symptomatic bradycardia with ventricular rate \<50 bpm), any ventricular arrhythmia, or other clinically significant heart disease. QTcF \>480 ms (Fridericia's formula), left ventricular ejection fraction (LVEF) \<50% by echocardiography at screening, or other significant electrocardiogram abnormalities.
• Malignancy within 5 years before signing the ICF, except for: curatively treated basal cell carcinoma of the skin, superficial bladder cancer, localized prostate cancer, biopsy-proven cervical carcinoma in situ or cervical squamous intraepithelial lesion detected by Pap smear, and completely resected ductal carcinoma in situ of the breast.
• Previous treatment with B-cell targeted therapies such as belimumab, rituximab, telitacicept; anti-CD22 agents (e.g., epratuzumab); anti-CD52 agents (e.g., alemtuzumab), or other similar biologics; TNF-α antagonists, IL-6 antagonists, IL-1 antagonists, selective T-cell costimulation modulators, etc., with less than 5 half-lives before screening.
• Use of any other investigational drug for SLE in a clinical trial within 4 weeks before screening.
• Presence of other serious diseases, including liver disease, neurological/psychiatric disorders, endocrine system disorders, hematological disorders (including moderate-to-severe dyslipidemia and related conditions), which in the investigator's judgment would affect participation in this study.
• Receipt of non-biologic investigational drugs (e.g., BTK inhibitors, JAK inhibitors), intravenous immunoglobulin, or plasma exchange within 4 weeks or 5 half-lives (whichever is shorter) before screening.
• Vaccination within 30 days before the first dose of study drug.
• Prior treatment with mRNA-LNP or other LNP-based drugs within 2 years before the first dose of study drug.
• History of asthma, severe allergic reactions, or known allergy to any active or inactive ingredient of the study drug (including background therapy).

Sponsors & Collaborators

• Circunited BioPharma (Shenzhen) Co., Ltd.: lead
• Boji Medical Technology Co., Ltd.: collaborator

Study Sites (1)

Beijing GoBroad Boren Hospital

Beijing, Beijing Municipality, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Anemia, Hemolytic, Autoimmune; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Immune System Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Yajing Zhang, M.D.

CONTACT

+86 10-83605002; yajing_cart66@126.com

Teresa Yang

CONTACT

+86 13902947747; muyizi.yang@circunited.com

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: May 20, 2026
• First Posted: June 5, 2026
• Study Start: April 7, 2026
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): May 30, 2027
• Last Updated: June 5, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)