NCT06508346

Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV

Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory ANCA-Associated Vasculitis

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 12 (estimated)
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
• Sex: All
• Age: 5 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of childhood-onset refractory ANCA-Associated Vasculitis.

Detailed description

The anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis （AAV ）is one of the most devastating and potentially fatal autoimmune diseases, characterized by involvement of small blood vessels (arterioles, tiny arteries, tiny static veins, and capillaries) and the presence of ANCA. The disease lead to extensive damage in multiple organs and systems,such as pulmonary hemorrhage and rapidly progressive glomerulonephritis (RPGN),ultimately resulting in disability and even death.Children with AAV are particularly at risk of organ damage, especially to the kidneys, and tend to more serious than that in adults. Currently, the primary treatment for AAV relies on glucocorticoids and immunosuppressants to alleviate symptoms. However, due to the absence of a curative treatment, patients often require lifelong medication. In recent years, biological agents such as rituximab have been introduced for the treatment of AAV, but still cannot completely eliminate autoimmune B cells in the bone marrow, leading to unsatisfactory overall outcomes. Furthermore, stopping the drugs can lead to relapse, and there is still no cure for AAV, leaving patients facing the challenges of lifelong medication and an incurable disease. Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Clinical studies have demonstrated that targeted CD19 CAR-T cells hold significant therapeutic potential for SLE. These cells effectively slow down the pathological progression of SLE and can also effectively treat severe cases. Furthermore, targeted CD19 CAR-T cells are also expected to restore the immune system in SLE patients, potentially allowing them to discontinue lifelong medication and avoid serious long-term side effects of drugs like hormones and immunosuppressants. Studies have reported that CAR-T has a good therapeutic effect on a variety of autoimmune diseases such as systemic sclerosis and idiopathic inflammatory dermatomyositis.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of childhood-onset refractory AAV.

Conditions

• ANCA Associated Vasculitis
• CAR-T Cell Therapy

Interventions

Type	Name	Description
BIOLOGICAL	anti-CD19-CAR-T cells	Intravenous injection

Timeline

• Start date: 2024-08-01
• Primary completion: 2026-07-31
• Completion: 2027-07-31
• First posted: 2024-07-18
• Last updated: 2024-12-02

Locations

1 site across 1 country: China