A Clinical Study of AFN50 in the Treatment of Autoimmune Diseases

NCT07448298 | Recruiting Early Phase 1

• 1 other identifier: KY2026-003-002
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a single-arm, open-label, single-centre exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of AFN50 Injection in adult patients with B-cell-mediated refractory/replapased autoimmune diseases.

Conditions

Relapsed/Refractory Autoimmune Diseases

Keywords

Autoimmune Diseases

Outcome Measures

Primary Outcomes (1)

• Adverse Event

  The incidence and severity of adverse events determined to be related to AFN50 treatment, as assessed per CTCAE v5.0.

  3 months

Secondary Outcomes (5)

• In vivo CAR T cell generation

  Day -28 to 28 days

• B cell ratios and counts in peripheral blood

  Day -28 to 12 months

• Changes in the 2000 Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2000) relative to baseline in participants

  Day -28 through Month 12

• SLE Responder Index-4 (SRI-4)

  Day-28 to12 months

• Changes in the Physician's Global Assessment (PGA) relative to baseline

  Day-28 to12 months

Study Arms (1)

AFN50 treatment group

EXPERIMENTAL

Intravenous infusion therapy with a dose escalation design

Biological: AFN50 injection

Interventions

AFN50 injection BIOLOGICAL

Intravenous infusion therapy. AFN50 was developed using novel T-cell-targeted lipid nanoparticles (T-LNP) that encapsulate RNA encoding a Chimeric Antigen Receptor.

AFN50 treatment group

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 to 69 years (inclusive), any gender. 1.3 Adequate bone marrow, coagulation, cardiac, pulmonary, hepatic, and renal function at screening:
• Bone Marrow Function:
• Absolute neutrophil count (ANC) ≥1.0 × 10⁹/L (no use of granulocyte colony-stimulating factor (G-CSF) within 7 days before screening; for long-acting G-CSF, a 14-day interval is required); ② Haemoglobin (Hb) ≥90 g/L (no red blood cell transfusion within 14 days before screening; use of recombinant human erythropoietin is permitted); ③ Platelet count (PLT) ≥75 × 10⁹/L; absolute lymphocyte count (ALC) ≥0.5 × 10⁹/L.
• Coagulation Function: International normalised ratio (INR) or activated partial thromboplastin time (APTT) ≤1.5 × the upper limit of normal (ULN).
• Cardiac Function: Left ventricular ejection fraction (LVEF) ≥50% as shown by echocardiography (ECHO).
• Pulmonary Function: Dyspnea ≤ CTCAE Grade 1, and pulse oxygen saturation (SpO₂) \>92% on room air.
• Hepatic Function: Alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≤2.5 × ULN; total bilirubin ≤1.5 × ULN.
• Renal Function: Creatinine clearance (Cockcroft-Gault formula) ≥50 mL/min, without requiring fluid support.
• Baseline oxygen saturation \>92% without supplemental oxygen. 1.5 Non-pregnant/non-lactating subjects. Women of childbearing potential must have a negative serum or urine pregnancy test report (women who have undergone surgical sterilisation or are postmenopausal for at least 2 years are not considered to be of childbearing potential) and must be willing to use contraception for 12 months following drug infusion.
• Relapsed/Refractory Systemic Lupus Erythematosus (SLE) 1.1 Meet the 2019 European Alliance of Associations for Rheumatology/American College of Rheumatology (EULAR/ACR) classification criteria for SLE.
• SLEDAI-2K score ≥6; if the score includes low complement and/or anti-dsDNA antibodies, the SLEDAI-2K clinical symptom score after excluding these two items must be ≥4.
• History of SLE for at least 6 months, with disease remaining active or relapsing despite receiving stable standard therapy for at least 8 weeks (drug doses stable for the past 2 weeks).
• Oral glucocorticoids (prednisone or equivalent) at a daily dose ≥7.5 mg and ≤30 mg; if combined with immunosuppressants, there is no minimum daily dose requirement.
• At least two immunosuppressants (including hydroxychloroquine) have been used in a standardised manner.
• Screening tests meet: positive serum antinuclear antibody (ANA), and/or positive anti-double-stranded DNA (anti-dsDNA) antibody, and/or hypocomplementemia (low C3 and/or low C4).

You may not qualify if:

• Presence of other uncontrolled active infections. 1.3 History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation.
• Received any mRNA-LNP product or other LNP-based therapy within the past 2 years and has a history of allergy to LNPs or their components.
• Received live vaccination within the past 30 days. 1.6 History of any severe cardiovascular disease within 6 months prior to screening, including: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmia, any ventricular arrhythmia, or other clinically significant cardiac disease.
• Pregnant or lactating women. 1.8 Individuals with asthma or a history of severe allergies. 1.9 According to the investigator's judgment, the individual is unlikely to complete all protocol-required study visits or procedures, including follow-up or compliance with study participation requirements.
• Other unspecified reasons that, in the opinion of the investigator, render the patient unsuitable for enrollment.
• Relapsed/Refractory Systemic Lupus Erythematosus: Concurrent neuropsychiatric lupus; thrombotic thrombocytopenic purpura (TTP)/microangiopathy (TMA).
• Relapsed/Refractory Sjögren's Syndrome: Concurrent liver cirrhosis; concurrent aplastic anaemia (AA), myelodysplastic syndrome (MDS), or other myeloproliferative disorders (MPD); drug-induced thrombocytopenia; TTP/TMA.
• Relapsed/Refractory Myasthenia Gravis: Presence of an uncontrolled myasthenic crisis within 2 weeks prior to screening.
• Relapsed/Refractory or Progressive Diffuse Cutaneous Systemic Sclerosis: NYHA Class IV cardiac function; presence of moderate to severe pulmonary hypertension (mean pulmonary artery pressure \>40 mmHg by echocardiography); FVC \<45% of predicted value; DLCO \<40% of predicted value; significant abnormalities on HRCT not attributable to SSc; persistent unexplained hematuria (\>5 red blood cells per high-power field) or creatinine clearance \<40 mL/min; prior history of autologous stem cell transplantation; signs of renal crisis; active gastric antral vascular ectasia.
• Relapsed/Refractory ANCA-Associated Vasculitis:
• Estimated glomerular filtration rate (eGFR) \<15 mL/min/1.73 m²; if the participant has alveolar haemorrhage requiring invasive mechanical ventilation expected to last beyond the screening period; requiring dialysis or plasma exchange during the screening period; or prior kidney transplantation.

Sponsors & Collaborators

• Beijing Boren Hospital: lead

Study Sites (1)

Beijing GoBroad Boren Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Autoimmune Diseases

Condition Hierarchy (Ancestors)

Immune System Diseases

Central Study Contacts

Yajing Zhang, Dr.

CONTACT

+8601083605002(716); 23975701@qq.com

Study Design

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

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: March 4, 2026
• Study Start: March 9, 2026
• Primary Completion (Estimated): February 29, 2028
• Study Completion (Estimated): February 28, 2029
• Last Updated: March 11, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)