A Study on the Safety, Preliminary Efficacy, and Cellular Kinetics of Allogeneic CD7-Targeted CAR-T Cell Injection for the Treatment of Type 1 Diabetes Mellitus
Type 1 diabetes mellitus (T1DM) is a T cell-mediated autoimmune disease characterized by autoimmune destruction of pancreatic beta cells, leading to absolute insulin deficiency and lifelong dependence on exogenous insulin. The disease results from loss of immune tolerance, with autoreactive T-cell responses against beta-cell antigens, and is typically associated with islet autoantibodies and insulitis. Although insulin therapy remains the standard of care, it does not correct the underlying autoimmune process. Non-insulin therapeutic strategies for T1DM are mainly directed toward immunomodulation and beta-cell replacement or regeneration. Among immunomodulatory approaches, previous studies have primarily focused on regulation of effector T cells and B cells. Novel immune-based therapies are needed to explore whether modulation of pathogenic immune cell populations may alter disease activity and preserve residual beta-cell function. The purpose of this study is to evaluate the safety, preliminary efficacy, and cellular kinetics of an allogeneic CD7-targeted CAR-T cell injection in participants with early stage T1DM. Participants will receive the investigational product and undergo regular assessments of safety, tolerability, treatment-emergent adverse events, cellular kinetics, glycemic parameters, exogenous insulin requirement, beta-cell function, and immunologic biomarkers. This study is expected to generate preliminary clinical evidence regarding the feasibility and potential therapeutic effects of CD7-targeted CAR-T cell therapy in T1DM.
• Age ≥18 years and ≤40 years.
• Participants with stage 2 or stage 3 type 1 diabetes mellitus, according to the staging criteria for type 1 diabetes defined in the ADA 2024 Standards of Care in Diabetes.
• Positive for at least one islet autoantibody at screening, including glutamic acid decarboxylase autoantibody (GADA), insulinoma-associated protein 2 autoantibody (IA-2A), insulin autoantibody (IAA) (applicable only to participants who have received insulin therapy for no more than 2 weeks), zinc transporter 8 autoantibody (ZnT8A), or islet cell autoantibody (ICA). Participants positive for two or more autoantibodies will be prioritized for enrollment.
• Peak C-peptide \>0.2 nmol/L during a mixed-meal tolerance test (MMTT), or fasting C-peptide \>0.1 nmol/L.
• The participant or his/her legally authorized representative voluntarily agrees to participate in the study and is able to sign the informed consent form.