Ignite Creation Date:
2025-12-24 @ 5:01 PM
Ignite Modification Date:
2025-12-29 @ 12:27 AM
Study NCT ID:
NCT07184450
Status:
RECRUITING
Last Update Posted:
2025-09-19
First Post:
2025-09-05
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases
Sponsor:
Chongqing Precision Biotech Co., Ltd
Organization:
Study Overview
Official Title:
Clinical Study of BCMA/CD70 Targeting Chimeric Antigen Receptor T Lymphocytes(CAR-T) in the Treatment of Refractory Pediatric Rheumatic Diseases
Status:
RECRUITING
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: