Efficacy and Safety of HN2301 in Autoimmune Diseases（AIDs）

NCT06801119 | Recruiting Phase 1

• 1 other identifier: HN2301-01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open lable and single arm study, is designed to evaluate the safety and preliminary efficacy of HN2301 in Autoimmune Disease（AID）

Conditions

Systemic Lupus Erythematosus; Scleroderma; Rheumatoid Arthritis

Keywords

SLE, SSc,RA

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs)

  Incidence, nature, and severity of treatment-emergent adverse events, assessed according to the study protocol and applicable toxicity grading criteria.

  Up to 3 months

Secondary Outcomes (14)

• vivo CAR T cell production

  Day-28 to14 days

• B cell ratio and counts in peripheral blood

  Day-28 to12 months

• Change from baseline of SLEDAI-2K score after HN2301 administration.

  Day-28 to12 months

• Quantify the clinical activity of HN2301 in patients using Physician Global Assessment (PGA) .

  Day-28 to12 months

• Proportion of participants achieving lupus low disease activity status (LLDAS)

  Day-28 to12 months

Study Arms (1)

HN2301 treatment group

EXPERIMENTAL

Participants will receive HN2301 Injection at the specified dose level and on the specified study days.

Drug: HN2301 injection

Interventions

HN2301 injection 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 cart

HN2301 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:

• Patients aged between 18 and 69 (inclusive), of any gender;
• Appropriate bone marrow, coagulation, cardiopulmonary, liver, and kidney functions. Bone marrow function: ANC ≥1.5×10\^9/L, ALC ≥0.8×10\^9/L, Hb ≥80g/L. No use of transfusions and growth factors allowed within 7 days prior to screening to meet these requirements. Coagulation function: INR or APTT ≤1.5×ULN. Cardiac function: Echocardiography (ECHO) assessment of left ventricular ejection fraction (LVEF) ≥40%. Lung function: ≤CTCAE grade 1 dyspnea and SpO2 ≥92% (measured by pulse oximetry) while breathing indoor air. Liver function: ALT and AST ≤2.5×ULN, total bilirubin \<2.0mg/dL (Gilbert syndrome subjects total bilirubin \<3.0mg/dL). Kidney function: defined as creatinine clearance rate (Cockcroft-Gault) ≥50mL/min without need for fluid assistance;
• Non-pregnant/non-lactating participants, willing to adopt contraceptive measures within 12 months after drug infusion;
• Diagnosed with SLE according to the 2019 EULAR/ACR SLE diagnostic criteria; A history of SLE for at least 6 months, having used a stable standard treatment regimen for at least 8 weeks; Oral corticosteroids are prednisone (or equivalent drug) ≥7.5mg/day and ≤30mg/day. At least two immunosuppressants have been used in a standardized manner (including hydroxychloroquine); Screening period tests meet: positive blood antinuclear antibody (ANA), and/or positive anti-ds-DNA antibodies, and/or hypocomplementemia;
• SSc-meets the classification criteria of ACR and EULAR, 10-35 in mRSS score;
• RA-meets the classification criteria of ACR and EULAR, DAS28-ESR\>3.2, ACPA possitive.

You may not qualify if:

• 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;
• Presence of other uncontrolled active infections;
• History of major organ transplantation (such as heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
• Pregnant or breastfeeding women;
• Receiving any mRNA-LNP product or other LNP medications within the past two years;
• 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;
• History of live vaccine administration within the last 30 days;
• Individuals with asthma, severe allergies;
• Other conditions deemed inappropriate for participation in this clinical study by the investigator.

Sponsors & Collaborators

• Shenzhen MagicRNA Biotechnology Co., Ltd: lead
• The First Affiliated Hospital of University of Science and Technology of China: collaborator

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China

Hefei, Anhui, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Scleroderma, Diffuse; Arthritis, Rheumatoid

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Scleroderma, Systemic; Skin Diseases; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Zhu Chen, MD

  The First Affiliated Hospital of University of Science and Technology of China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zhu Chen, MD

CONTACT

+86055162284920; doczchen@ustc.edu.cn

Ze Xiu Xiao, MD

CONTACT

+86075527109036; xiaozexiu@magicrna.com

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: January 15, 2025
• First Posted: January 30, 2025
• Study Start: March 16, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): June 30, 2028
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)