Neuromyelitis Optica Spectrum Disorder Clinical Trial
Official title:
Single-center, Open Label Trial of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder
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.
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 steroids,azathioprine 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.
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