Conditions: Neuromyelitis Optica Neuromyelitis Optica Spectrum Disorder
Keywords: NMO NMOSD NMO-IgG Aquaporin-4 AQP4 anti-AQP4
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 10
Healthy volunteers: no

Brief summary:
This is a phase 1b interventional trial of bevacizumab (Avastin®) to evaluate the tolerability/safety and preliminary efficacy of bevacizumab (Avastin®) as add-on therapy for treatment of acute optic neuritis and/or transverse myelitis in neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD). A single infusion of Avastin® is added to standard-of-care high dose steroids and an additional dose of Avastin® is added to plasma exchange (if necessary). The primary outcomes are clinical changes in the Expanded Disability Severity Scale, Timed 25-foot Walk and Low Contrast Visual Acuity, MRI parameters and safety.

Detailed description:
Study Objective: The overall objective is to evaluate the tolerability/safety and efficacy of adding bevacizumab (Avastin®) to standard of care therapy in improving clinical and radiologic outcomes of acute optic neuritis and/or transverse myelitis in neuromyelitis optica and neuromyelitis optica spectrum disorders.

Primary Objective: To compare the clinical and radiographic outcome following acute optic neuritis and/or transverse myelitis in NMO/NMOSD in patients who receive 1-2 doses of 10 mg/kg dose of bevacizumab (Avastin®) in addition to standard medical therapy.

Secondary Objectives:

* To determine the effect of Avastin on NMO clinical scores (Expanded Disability Status Scale, Timed 25-foot Walk and Low Contrast Visual Acuity \[LCVA\]).
* To evaluate the safety and tolerability of a 10 mg/kg dose of intravenous Avastin.
* To determine the frequency of adverse events with Avastin in this patient population.
* To determine the effect of Avastin on MRI lesion size and extent.

The duration of the investigation is 1-2 years.

Arms:
arm 1: Bevacizumab 10 mg/kg intravenous infusion at onset of exacerbation and, if needed, a second time during the plasma exchange phase.

Interventions: intervention 1: Bevacizumab
Intervention descriptions: intervention 1: None

Protocol document: