NCT06792344

Clinical Study of CD19 CAR-T in the Treatment of Refractory Systemic Sclerosis

Clinical Study of CD19 Targeting Chimeric Antigen Receptor T Lymphocytes (CAR-T) in the Treatment of Refractory Systemic Sclerosis

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
• Sex: All
• Age: 5 Years – 18 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 systemic sclerosis

Detailed description

Systemic sclerosis (SSc) is a type of systemic autoimmune disease characterized by skin thickening and hardening. In addition to skin involvement, it can also affect the internal organs (lungs, cardiovascular, kidney, digestive tract, etc.). Currently there are no drugs that can affect the natural course of systemic sclerosis, and individualized treatment according to target organ involvement is usually used, glucocorticoid is used for patient combined with myositis or mixed connective tissue , and various immunosuppressants, including azathioprine, mycophenolate and cyclophosphamide, may be helpful in the treatment of interstitial lung disease. The anti-IL-6 blocker tocilizumab and the anti-fibrotic drug nitidanib have also been shown to be effective to SSC-associated interstitiallungdisease (ILD) and cardiac involvment. 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 SSc.

Conditions

• Systemic Sclerosis (SSc)

Interventions

Type	Name	Description
OTHER	anti-CD19 CAR-T cells	Intravenous injection the targeting CD19 CAR T cell
DRUG	anti-CD19 CAR-T cells	CD19-targeting CAR-T cells

Timeline

• Start date: 2024-12-15
• Primary completion: 2027-12-30
• Completion: 2027-12-30
• First posted: 2025-01-24
• Last updated: 2025-12-22

Locations

2 sites across 2 countries: China, Christmas Island