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A Study of BEN301 Injection in the Treatment of Autoimmune Diseases

ConditionSSC RA SS IIM
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-70
SponsorBeijing Boren Hospital

About this trial

The study aims to investigate the safety, tolerability, and preliminary clinical efficacy of BEN301 Injection in patients with autoimmune diseases.

In patients with autoimmune diseases, Treg cells are typically deficient or dysfunctional. CAR-Treg cell therapy represents a promising strategy for the treatment of autoimmune diseases and may be applicable to a broad spectrum of autoimmune conditions. Preclinical studies have shown that BEN301Injection not only effectively suppresses the aberrant activation of T and B cells in SLE models, but also significantly reduces total lgG secretion and the production of SLE-specific autoantibodies, particularly anti-double-stranded DNA (dsDNA) antibodies. Moreover, no significant treatment-related toxicities were observed.

Treg cell therapy has already demonstrated favorable efficacy in multiple indications, including organ transplantation and autoimmune diseases, with a well-established safety and tolerability profile. Meanwhile, CAR-Treg cell therapy has been actively explored in various autoimmune conditions; several products have shown therapeutic potential, and some patients have already benefited from treatment. These findings highlight the promising prospects of CAR-Treg cell therapy in the management of autoimmune diseases.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

General Requirements

Voluntary participation in the study, providing written informed consent, and willingness and ability to comply with the protocol.

Age 18-70 years (inclusive) at the time of signing informed consent.

Expected survival ≥ 12 months.

Disqualifiers

Severe Renal Disease

History of severe nephritis or nephrotic syndrome.

Severe Cardiovascular Disease

History of severe cardiovascular disease, including but not limited to: symptomatic chronic heart failure requiring intervention, acute myocardial infarction or coronary artery bypass grafting within 6 months, unstable angina, history of severe arrhythmia, or history of stroke.

Trial design

Design model

Sequential

Treatments tested in this trial

  • CD4⁺ CAR⁺ Foxp3⁺ cells

    Biological/Vaccine

    Single administration of 1×10⁸ viable CD4⁺ CAR⁺ Foxp3⁺ cells. Lymphodepletion is generally not required; however, if Treg expansion is suboptimal, lymphodepletion may be administered as needed, or low-dose IL-2 may be combined. The recommended lymphodepletion regimens 33 include: * Cyclophosphamide monotherapy (CTX 900-1000 mg/m² on day -3, or CTX 300 mg/m² from day -5 to day -3); * or cyclophosphamide combined with fludarabine (12.5-25 mg/m² from day -5 to day -3); * or low-dose IL-2 administered after cell infusion.

  • CD4⁺ CAR⁺ Foxp3⁺ cells

    Biological/Vaccine

    Single administration of 3×10⁸ viable CD4⁺ CAR⁺ Foxp3⁺ cells. Lymphodepletion is generally not required; however, if Treg expansion is suboptimal, lymphodepletion may be administered as needed, or low-dose IL-2 may be combined. The recommended lymphodepletion regimens 33 include: * Cyclophosphamide monotherapy (CTX 900-1000 mg/m² on day -3, or CTX 300 mg/m² from day -5 to day -3); * or cyclophosphamide combined with fludarabine (12.5-25 mg/m² from day -5 to day -3); * or low-dose IL-2 administered after cell infusion.

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: BEN301 Low-dose groupExperimental treatment 1 intervention
Group B: BEN301 High-dose groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

DLT

Proportion of participants experiencing dose-limiting toxicity (DLT) within 28 days after infusion; determination of the MTD or RP2D

Time frame
days 28
2

AE

Incidence and proportion of adverse events occurring after infusion.

Time frame
AEs occurring from informed consent signing through Week 12 post-infusion. Thereafter, only AEs related to the investigational product will be collected through study completion, an average of 2 years.

Secondary outcomes

1

Modified Rodnan Skin Score (mRSS)

Change from baseline in modified Rodnan Skin Score (mRSS) at Week 12 after the first infusion of BEN301 Injection. The minimum value of the modified Rodnan skin scale (MRSS) is 0 points, and the maximum value is 51 points. The higher the score, the more severe the degree of skin sclerosis, that is, the worse the result. MRSS quantifies the skin involvement of systemic sclerosis by evaluating the skin hardness of 17 parts of the body. 0 points represent normal skin and 3 points represent severe thickening. The higher the total score, the more extensive and severe skin fibrosis, which is associated with disease activity and poor prognosis.

Time frame
Week 12
2

Simplified Disease Activity Index (SDAl) score

SDAl score at Week 12 after the first infusion of BEN301 Injection; percentage of patients achieving SDAl low disease activity and clinical remission. SDAI score exceeding 26 is considered high disease activity; exceeding 11 and not greater than 26, moderate disease activity; exceeding 3.3 and not greater than 11, low disease activity.

Time frame
Week 12
3

EULAR Sjogren's Syndrome Disease Activity Index (ESSDAl) score

Change from baseline in overall disease activity (ESSDAl) at Week 12 after the first infusion of BEN301 Injection. The ESSDAI is a validated tool used in clinical studies to measure the systemic disease activity in participants with primary Sjogren's syndrome. The ESSDAI includes 12 domains divided into 4 activity levels, where zero represents no activity. A higher score means a worse outcome.

Time frame
Week 12
4

Total Improvement Score (TIS)

Change from baseline in Myositis Response Criteria TIS scores at Week 12 after the first infusion of BEN301 Injection. The Total Improvement Score (TIS) is a standardized composite measure developed to quantify treatment response in idiopathic inflammatory myopathies(IIM). The TIS ranges from 0 to 100,with higher scores reflecting greater improvement.

Time frame
Week 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Beijing Boren Hospital

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: