Clinical Study of AFN50 Injection in the Autoimmune Diseases

This is an investigator-initiated trial designed to evaluate the safety, tolerability and primary efficacy of AFN50 injection for the treatment of autoimmune diseases.

Study Overview

• Status: Recruiting
• Conditions: Autoimmune Diseases
• Intervention / Treatment: Biological: AFN50 injection

Detailed Description

This study is a prospective exploratory clinical trial in subjects with autoimmune diseases, mainly relapsing and refractory systemic lupus erythematosus. The objective is to evaluate the safety, tolerability, and primary efficacy of AFN50 injection in relapsing and refractory systemic lupus erythematosus.

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Beicheng SUN, Dr; Phone Number: +86 551 6292 2800; Email: sunbc0207@163.com
• Study Contact Backup: Name: Huan ZHOU, Dr; Phone Number: +8613665527160; Email: zhouhuanbest@vip.163.com

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230001
      • Recruiting
      • First Affiliated Hospital of Anhui Medical University
      • Contact: Huan Zhou, PhD; Phone Number: +86 055162922800; Email: zhouhuanbest@vip.163.com
      • Principal Investigator: Beichen Sun, PhD
      • Principal Investigator: Huan Zhou, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Be able to understand and voluntarily sign the written informed consent form;
2. Patients aged between 18 and 69 (inclusive), of any gender, diagnosed with SLE according to the 2019 EULAR/ACR SLE diagnostic criteria;
3. A history of SLE for at least 6 months, having used a stable standard treatment regimen for at least 8 weeks, with the dosage stable for 2 weeks, yet the disease remains active or has relapsed; Standard treatment refers to the stable use of the following drugs alone or in combination: non-steroidal anti-inflammatory drugs (NSAIDs), antimalarials, corticosteroids; immunosuppressants (including but not limited to cyclophosphamide, methotrexate, azathioprine, mycophenolate mofetil, leflunomide, tacrolimus, cyclosporine); targeted drugs (including but not limited to belimumab, telitacicept, eculizumab, rituximab);
4. Oral corticosteroids are prednisone (or equivalent drug) ≥7.5mg/day and ≤30mg/day. If used in combination with immunosuppressants, there is no minimum daily dose requirement;
5. Standardized treatment failure with hydroxychloroquine or at least two immunosuppressants;
6. Screening period tests meet: positive blood antinuclear antibody (ANA), and/or positive anti-double-stranded DNA (anti-dsDNA) antibodies, and/or hypocomplementemia (low C3 and/or C4);
7. Screening period SLEDAI-2K score ≥6 points. If scoring includes low complement and/or anti-ds-DNA antibodies, the score for SLEDAI-2K clinical symptoms (excluding low complement and/or anti-ds-DNA antibodies) should be ≥4 points;

8. Appropriate bone marrow, coagulation, cardiopulmonary, liver, and kidney functions.

   Bone marrow function: Absolute Neutrophil Count (ANC) ≥1.5×10⁹/L (no granulocyte colony-stimulating factor (G-CSF) administered within 7 days prior to screening; a 14-day interval required for long-acting G-CSF); Hemoglobin (Hb) ≥80 g/L (no red blood cell transfusion within 14 days prior to screening; recombinant human erythropoietin is permitted. For patients meeting the Hb ≥80 g/L inclusion criterion, red blood cell transfusion is allowed during treatment to maintain hemoglobin level at ≥80 g/L); Platelet Count (PLT) ≥50×10⁹/L, Absolute Lymphocyte Count (ALC) ≥0.8×10⁹/L.

   Coagulation function: International Normalized Ratio (INR) or Activated Partial Thromboplastin Time (APTT) ≤1.5 times the upper limit of normal (ULN).

   Cardiac function: Left Ventricular Ejection Fraction (LVEF) ≥40% as measured by echocardiography (ECHO).

   Pulmonary function: Dyspnea of ≤CTCAE Grade 1; pulse oxygen saturation (SpO2) >92% under room air.

   Hepatic function: Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) ≤2.5×ULN; total bilirubin ≤1.5×ULN.

   Renal function: Creatinine clearance rate (calculated by the Cockcroft-Gault formula) ≥50 mL/min, without the need for fluid support;

9. Baseline oxygen saturation >92% without oxygen supplementation;
10. Non-pregnant/non-lactating participants. Women of childbearing potential must have a negative serum or urine pregnancy test result (women who have undergone surgical sterilization or postmenopausal women for at least 2 years are not considered women of childbearing potential) and be willing to adopt contraceptive measures within 12 months after drug infusion.

Exclusion Criteria:

1. Individuals with positive Hepatitis B surface antigen (HBsAg) and/or Hepatitis B core antibody (HBcAb), and Hepatitis B virus (HBV) DNA positivity or titers above the detection threshold; those with positive Hepatitis C virus (HCV) antibodies and HCV RNA positivity or titers above the detection threshold; individuals with Human Immunodeficiency Virus (HIV) antibodies positivity, CMV DNA positivity or above the detection limit; those with positive syphilis antigen or antibodies;
2. Presence of other uncontrolled active infections;
3. History of major organ transplantation (such as heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
4. Receiving any mRNA-LNP product or other LNP medications within the past two years, and with a history of allergy to LNP and its components;
5. History of live vaccine administration within the last 30 days;
6. History of any of the following cardiovascular diseases within the last 6 months before screening: Class III or IV heart failure defined by the New York Heart Association (NYHA), myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant cardiac diseases;
7. Pregnant or breastfeeding women;
8. Individuals with asthma, severe allergies;
9. In the investigator's judgment, the participate is unlikely to complete all protocol-required study visits or procedures, including follow-up visits or adherence to the study participation requirements.
10. Other conditions deemed inappropriate for participation in this clinical study by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Participant Group; AFN50 Injection	Biological: AFN50 injection; Intravenous infusion therapy. AFN50 was developed using novel T-cell-targeted lipid nanoparticles (T-LNP) encapsulating mRNA encoding Chimeric Antigen Receptor.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events	Incidence and severity of AEs associated with AFN50 as assessed by CTCAE v5.0	3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
in vivo CAR T cell production	The counts, proportions and sustained days of CAR-T cells in the peripheral blood	Day-28 to 28 days
B cell ratios and counts in peripheral blood	Assessment of the change of B cell ratios and counts in peripheral blood after AFN50 treatment	Day-28 to 12 months
Changes in the 2000 Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2000) relative to baseline in participants	Assessment of Systemic Lupus Erythematosus Disease Activity Index 2000 from baseline to the month 12 follow-up visit. A total score can fall between 0 and 105, which determines changes in the disease activity of patients.	Day-28 to12 months
SLE Responder Index-4 (SRI-4)	Achievement of SRI-4 response at one or more scheduled study visits between baseline and the Month 12 follow-up.	Day-28 to12 months
Changes in the Physician's Global Assessment (PGA) relative to baseline	A total score can range from 0.0 to 3.0, with higher scores indicating more severe disease activity.	Day-28 to12 months
Proportion of participants achieving DORIS remission	Proportion of participants achieving DORIS (Definition Of Remission In SLE) remission after AFN50 administration.	Day-28 to12 months
Proportion of participants achieving complete renal response (CRR)	Proportion of participants achieving complete renal response (CRR) after AFN50 administration	Day-28 to12 months
Proportion of participants achieving low disease activity status (LLDAS)	Maintenance of LLDAS: The proportion of participants with SLE who remain in Lupus Low Disease Activity State at predefined study visits through the 12-month follow-up period.	Day-28 to12 months

Collaborators and Investigators

• Sponsor: AlphaNa Bioscience Company Limited
• Collaborators: The First Affiliated Hospital of Anhui Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): February 4, 2026
• Primary Completion (Estimated): February 4, 2028
• Study Completion (Estimated): February 3, 2029

Study Registration Dates

• First Submitted: February 4, 2026
• First Submitted That Met QC Criteria: February 10, 2026
• First Posted (Actual): February 13, 2026

Study Record Updates

• Last Update Posted (Actual): March 5, 2026
• Last Update Submitted That Met QC Criteria: March 3, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: SLE; AFN50
• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases
• Other Study ID Numbers: PJ-2025-12-57

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No