NCT07184450

Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases

Clinical Study of BCMA/CD70 Targeting Chimeric Antigen Receptor T Lymphocytes(CAR-T) in the Treatment of Refractory Pediatric Rheumatic Diseases

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (estimated)
• Sponsor: Chongqing Precision Biotech Co., Ltd · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This is an investigator-initiated trial to evaluate the efficacy and safety of BCMA/CD70-targeted CAR-T in the treatment of refractory pediatric rheumatic diseases.

Detailed description

Pediatric rheumatic diseases include juvenile dermatomyositis（JDM）, polyarticular juvenile idiopathic arthritis, systemic sclerosis（SSc）, primary Sjogren's syndrome, etc. Juvenile dermatomyositis (JDM) is a non-suppurative chronic autoimmune disease and the most common type of juvenile idiopathic inflammatory myopathy. Its clinical manifestations include proximal limb muscle weakness and various rashes, which may be accompanied by multiple system involvement such as respiratory, cardiovascular, digestive and nervous systems.Juvenile idiopathic arthritis (JIA) is mainly characterized by chronic synovitis of the joints and also may be accompanied by functional damage to multiple organs. It is an important cause of disability and blindness in childhood. Polyarticular type is a relatively common subtype of JIA, with severe joint symptoms. Systemic sclerosis (SSc) is characterized by thickened and hardened skin and can also affect internal organs. Sjogren's syndrome (SS) is a systemic autoimmune disease that affects exocrine glands. The clinical manifestations of SS in children are mainly recurrent parotitis, parotid gland swelling and systemic symptoms.Despite the active treatment measures, these patients are still intolerant or unresponsive to the treatment, resulting in a high disability and mortality rate. CAR-T therapy is an adoptive cell therapy that uses genetic modification technology to reprogram T cells and eliminate target cells expressing disease-related antigens through antigen-specific recognition.Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases.Many kinds of autoimmune diseases such as systemic lupus erythematosus，systemic sclerosis, idiopathic inflammatory myopathy have good therapeutic effect. These results suggest that CAR-T cells may play a role in several different B-cell-mediated and autoantibody-driven human autoimmune diseases, which is expected to bring breakthroughs in the treatment of refractory pediatric rheumatic diseases.

Conditions

• Juvenile Dermatomyositis (JDM)
• Polyarticular Juvenile Idiopathic Arthritis
• Systemic Sclerosis (SSc)
• Primary Sjogren's Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	BCMA/CD70-targetd CAR-T	Subjects underwent lymphocytetion cheotherapy and then received a single intravenous cell infusion

Timeline

• Start date: 2025-09-01
• Primary completion: 2026-03-30
• Completion: 2028-09-30
• First posted: 2025-09-19
• Last updated: 2025-09-19

Locations

1 site across 1 country: China