Dose-escalation Study to Assess the Safety, Tolerability, and Preliminary Efficacy of HN2301 in Patients With Autoimmune Diseases Including Systemic Lupus Erythematosus（SLE）, Systemic Sclerosis (SSc) and Rheumatoid Arthritis （RA）

Plain English Summary

Efficacy and Safety of HN2301 in Autoimmune Diseases（AIDs） is a Phase 1 clinical trial sponsored by Shenzhen MagicRNA Biotechnology Co., Ltd studying Systemic Lupus Erythematosus, Scleroderma, Rheumatoid Arthritis. This trial tests a new drug called HN2301 for people with autoimmune diseases. It is for adults aged 18-69 with active Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), or Rheumatoid Arthritis (RA) that hasn't responded well to current treatments. Participants will receive HN2301, and their safety and how well the drug works will be closely monitored. Standard treatments for these conditions are available, but this trial explores a new option. The trial aims to enroll 30 participants.

Official 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）

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 to 69 with a diagnosed and active form of Lupus, Scleroderma, or Rheumatoid Arthritis. Patients must have had their condition for at least 6 months and have tried standard treatments that weren't fully effective. You cannot join if you have certain infections (like Hepatitis B, C, HIV, syphilis), have had an organ transplant, are pregnant or breastfeeding, or have had recent mRNA-LNP treatments. Specific health requirements for heart, lung, liver, kidney, and blood function must be met. This trial is studying Systemic Lupus Erythematosus, Scleroderma, Rheumatoid Arthritis, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how often participants experience side effects during the first three months, which helps determine the drug's safety. The specific primary outcome measures are: Incidence of treatment-emergent adverse events (TEAEs) (Up to 3 months). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 1, the first major stage of clinical testing. Phase 1 trials typically involve 20-100 participants and focus on safety, dosage levels, and side effects. The primary goal is not to test whether the treatment works but to establish that it is safe enough for further testing. About 70% of Phase 1 drugs advance to Phase 2. If successful, the treatment will proceed to Phase 2 efficacy testing.

Why This Trial Matters

This trial is important because it investigates a novel treatment for debilitating autoimmune diseases, addressing a need for new therapies when existing options are insufficient. This research targets Systemic Lupus Erythematosus, Scleroderma, Rheumatoid Arthritis, where improved treatment options are needed.

Investor Insight

This early-phase trial by a biotechnology company signals a potential new entrant in the autoimmune disease market, with a focus on novel RNA-based therapies. Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the specific risks and benefits of HN2301 and how it compares to your current treatment. Participation involves receiving the study drug and attending regular appointments for monitoring of safety and disease activity. You will need to use effective birth control for 12 months after receiving the drug if you are of childbearing potential. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 30 participants

Interventions

• DRUG: HN2301 injection — 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.

Primary Outcomes

• Incidence of treatment-emergent adverse events (TEAEs) (Up to 3 months)

Secondary Outcomes

• 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)

Full Eligibility Criteria

Inclusion Criteria:

1. Patients aged between 18 and 69 (inclusive), of any gender, diagnosed with SLE according to the 2019 EULAR/ACR SLE diagnostic criteria;
2. A history of SLE for at least 6 months, having used a stable standard treatment regimen for at least 8 weeks, with the dosage stabilized 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 cyclophosphamide, methotrexate, azathioprine, mycophenolate mofetil, leflunomide, tacrolimus, cyclosporine, etc.; targeted drugs include belimumab, rituxima, eculizumab, rituximab, etc.
3. 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;
4. At least two immunosuppressants have been used in a standardized manner (including hydroxychloroquine);
5. Screening period tests meet: positive blood antinuclear antibody (ANA), and/or positive anti-ds-DNA antibodies, and/or hypocomplementemia;
6. 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; 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.
7. 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;
8. Non-pregnant/non-lactating participants, 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. Pregnant or breastfeeding women;
5. Receiving any mRNA-LNP product or other LNP medications within the past two years;
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. History of live vaccine administration within the last 30 days;
8. Individuals with asthma, severe allergies;
9. Other conditions deemed inappropriate for participation in this clinical study by the investigator.

Trial Locations

• The First Affiliated Hospital of University of Science and Technology of China, Hefei, Anhui, China

Frequently Asked Questions

Related Conditions

• Systemic Lupus Erythematosus
• Systemic Sclerosis
• Rheumatoid Arthritis
• Autoimmune Diseases
• Immunology
• Inflammatory Diseases
• Connective Tissue Diseases
• Vasculitis
• Sjogren's Syndrome
• Dermatomyositis

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