ClinicalTrials.gov record NCT07629596

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)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

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 (Systemic Lupus Erythematosus and Autoimmune Hemolytic Anemia)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhaseEarly phase 1A very early study, usually in a small number of people, to learn how the body handles a treatment. It is not designed to show whether the treatment works.
Age18 years old to 60 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label, single-arm early Phase 1 study evaluates Arnovie101, an mRNA-LNP in vivo CAR‑T drug, in adults with relapsed/refractory SLE or refractory AIHA. Participants receive intravenous Arnovie101 at an initial low dose with possible escalation and are followed for safety, drug behavior, immune effects, and disease measures for up to 12 months. The study is exploratory and collects safety and biological activity data.

What it is studying. The study is testing the safety (including dose-limiting toxicities and adverse events), how the drug is processed in the body (pharmacokinetics), and its immune and disease effects (pharmacodynamics). It will measure B‑cell counts, in‑vivo CAR‑T production, cytokines and inflammatory markers, SLE disease activity scores, and hematologic responses in AIHA.

What the researchers hope to learn. To evaluate safety (incidence of dose-limiting toxicities and adverse events), describe pharmacokinetics and pharmacodynamics of Arnovie101, measure effects on B cells and in‑vivo CAR‑T production, assess changes in SLE disease activity scores and hematologic response in AIHA, and measure cytokines, inflammatory markers, immunogenicity (anti-drug antibodies), and levels of cationic lipids.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Arnovie101 Infusion Intravenous

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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).
  • Female participants must meet the following criteria: 1) Not pregnant or breastfeeding; 2) Surgically sterile or postmenopausal for ≥2 years, or if of childbearing potential (including those postmenopausal <2 years), have a negative serum pregnancy test (β-hCG) and agree to use effective contraception (e.g., condom, spermicide, or intrauterine device) during the study and for at least 12 months after the last dose. Use of progesterone-only contraceptives is not permitted; 3) Agree not to breastfeed during the study and for at least 12 months after the last dose.
  • Male participants must meet the following criteria: If not surgically sterile and engaging in sexual activity that could lead to pregnancy, agree to use effective contraception (e.g., condom, spermicide) during the study and for at least 12 months after the last dose, and refrain from donating semen or sperm during the study and for 12 months after the last dose.

Exclusion criteria

  • 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).
  • Prior CAR-T cell therapy.
  • Major surgery within 4 weeks before the first dose of study drug, or minor surgery within 2 weeks before the first dose.
  • Severe mental disorder or suicidal tendency.
  • Any other condition that, in the investigator's judgment, makes the participant unsuitable for this study.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This is a prospective, exploratory clinical trial of Arnovie101 injection, an mRNA-LNP-based in vivo CAR-T therapy, in subjects with B cell-mediated autoimmune diseases (SLE, AIHA). The objective is to evaluate its safety, PK, and PD in these conditions.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive an intravenous infusion of Arnovie101; dosing begins at a low level and may be escalated per the protocol. They will attend clinic visits for safety monitoring and study tests (blood draws for drug levels, B‑cell counts, CAR‑T detection, cytokines and other labs), and disease assessments such as SLEDAI‑2K or hematologic measures for AIHA. Monitoring and assessments continue up to 12 months after treatment. Participants must follow contraception and semen‑donation restrictions for 12 months after the last dose.

How the study is structured. Open-label, single-group (no placebo) early Phase 1 treatment study. Participants receive intravenous Arnovie101 infusions with a planned dose-escalation scheme. There is no masking. Follow-up includes regular clinic visits with blood tests, immune monitoring, disease activity assessments, and adverse-event monitoring for up to 12 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on autoimmune hemolytic anemia

Clinical trials49 trials listed for autoimmune hemolytic anemiaEvery trial the registry lists for autoimmune hemolytic anemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in autoimmune hemolytic anemia researchLatest monthly update: September 2026. Includes: Negative DAT cases. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 7, 2026
Primary completion
May 30, 2027 (estimated)
Study completion
May 30, 2027 (estimated)
Target enrollment
5 participants (estimated)
Last updated on registry
Jun 5, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Circunited BioPharma (Shenzhen) Co., Ltd.
Collaborators
  • Boji Medical Technology Co., Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

single arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
PK
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
relapsed/refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
standard therapy
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
immunosuppressants
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
relapse
A condition that improved with treatment and has since returned or worsened.
hydroxychloroquine
A medicine used for some autoimmune conditions. It appears in eligibility criteria when a study requires or excludes its use.
Life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
lines of therapy
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
Absolute neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
ULN
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
QTcF
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
echocardiography
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
biopsy-proven
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
5 half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
CAR-T
A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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