Ignite Creation Date:
2024-05-06 @ 9:04 AM
Last Modification Date:
2024-10-26 @ 12:09 PM
Study NCT ID:
NCT02893111
Status:
COMPLETED
Last Update Posted:
2024-04-11
First Post:
2016-08-27
Brief Title:
Efficacy and Safety of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
Sponsor:
Tianjin Medical University General Hospital
Organization:
Tianjin Medical University General Hospital
Study Overview
Official Title:
Single-center Open Label Trial of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
Status:
COMPLETED
Status Verified Date:
2024-04
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:
Neuromyelitis Optica Spectrum Disorders NMOSD is characterized by the pathogenic anti-AQP4 antibody which can be produced by specific plasma cells The patients who are not responsive to rituximab treatment may be due to the presence of short-lived and long-lived plasma cells Previous studies confirmed that the proteasome inhibitor bortezomib Velcade approved for therapy of multiple myeloma eliminated both plasmablasts and plasma cells by activation of the terminal unfolded protein response Treatment with bortezomib may help deplete plasma cells producing auto-antibodies Therefore the rationale for using bortezomib in NMOSD is in that bortezomib may help eliminate autoreactive plasma cells and reduce anti-AQP4 antibodies titers It is noted that bortezomib may protect astrocytes from NFκB-dependent inflammatory damage in early events in NMOSD pathogenesis
The purpose of this study is to determine if the drug bortezomib contributes to reduce the average relapsing rates ARRs and alleviate neurological disability in NMOSD patients
The purpose of this study is to determine if the drug bortezomib contributes to reduce the average relapsing rates ARRs and alleviate neurological disability in NMOSD patients
Detailed Description:
It has been shown in some scientific studies that the the antibody marker specific for neuromyelitis optica spectrum disorders NMOSD known as AQP4-IgG causes inflammation in brain tissues by activating NF-κB pathway Bortezomib has already been shown to be effective in systemic lupus erythematosus SLE
The overall objective is to assess the efficacy and safety of bortezomib as add-on therapy to oral steroidsazathioprine or others for treatment of relapsing NMOSD which have not reduced average relapsing rate ARR effectively
The primary most important objectives of this study are to determine
Whether bortezomib reduces relapse frequency in patients with relapsing NMO The number of attacks during the one year treatment period will be compared to the number of attacks that occurred prior to initiation of bortezomib treatment
The secondary objectives are to determine
The safety profile of bortezomib in patients with NMO Whether bortezomib maintains or improves walking visual function and quality of life as measured by a variety of established disability scales We will also assess the severity of an individual attack and the degree of recovery
Depending on our preliminary investigations we may evaluate patient cerebrospinal fluid in the laboratory to see how effective eculizumab is at getting into the cerebrospinal fluid from the blood stream and to see if the drug reverses the biological effects of the NMO-IgG antibody
The overall objective is to assess the efficacy and safety of bortezomib as add-on therapy to oral steroidsazathioprine or others for treatment of relapsing NMOSD which have not reduced average relapsing rate ARR effectively
The primary most important objectives of this study are to determine
Whether bortezomib reduces relapse frequency in patients with relapsing NMO The number of attacks during the one year treatment period will be compared to the number of attacks that occurred prior to initiation of bortezomib treatment
The secondary objectives are to determine
The safety profile of bortezomib in patients with NMO Whether bortezomib maintains or improves walking visual function and quality of life as measured by a variety of established disability scales We will also assess the severity of an individual attack and the degree of recovery
Depending on our preliminary investigations we may evaluate patient cerebrospinal fluid in the laboratory to see how effective eculizumab is at getting into the cerebrospinal fluid from the blood stream and to see if the drug reverses the biological effects of the NMO-IgG antibody
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