Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06508346
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-07-14
Brief Title:
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory Refractory AAV
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cells in Children With Refractory ANCA-Associated Vasculitis
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 systemssuch as pulmonary hemorrhage and rapidly progressive glomerulonephritis RPGNultimately resulting in disability and even deathChildren 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 dermatomyositisThe 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
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 dermatomyositisThe 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None