NCT07527936

A Study of BEN301 Injection in theTreatment of Autoimmune Diseases

A Phase I Exploratory Study on the Safety and Efficacy of BEN301 Injection in the Treatment of Autoimmune Diseases

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: RenJi Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

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 BEN301 Injection 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.

Conditions

• Systemic Sclerosis (SSc)
• Rheumatoid Arthritis (RA)
• Sjogren's Syndrome (SS)
• Idiopathic Inflammatory Myopathies(IIM)

Interventions

Type	Name	Description
BIOLOGICAL	Single administration of 1x10^8 viable CD4+ CAR+ Foxp3+ cells.	Lymphodepletion is generally not required; however, if Treg expansion issuboptimal, lymphodepletion may be administered as needed, or low-dose IL-2 may be combined. The recommended lymphodepletion regimens include: Cyclophosphamide monotherapy (CTX 900-1000 mg/m2 on day -3,or CTX 300 mg/m\^2 from day -5 to day -3); or cyclophosphamide combined with fludarabine (12.5-25 mg/m\^2 from day -5 to day -3); - or low-dose IL-2 administered after cell infusion.
BIOLOGICAL	Single administration of 3x10^8 viable CD4+ CAR+ Foxp3+ cells.	Lymphodepletion is generally not required; however, if Treg expansion issuboptimal, lymphodepletion may be administered as needed, or low-dose IL-2 may be combined. The recommended lymphodepletion regimens include: Cyclophosphamide monotherapy (CTX 900-1000 mg/m2 on day -3,or CTX 300 mg/m\^2 from day -5 to day -3); or cyclophosphamide combined with fludarabine (12.5-25 mg/m\^2 from day -5 to day -3); - or low-dose IL-2 administered after cell infusion.

Timeline

• Start date: 2026-04-20
• Primary completion: 2028-08-31
• Completion: 2029-02-28
• First posted: 2026-04-14
• Last updated: 2026-04-14

Locations

1 site across 1 country: China