An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica
The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica|
- Reduction in number of neuromyelitis optica (NMO)relapses, compared to before entering study. [ Time Frame: 15 months ] [ Designated as safety issue: No ]
- Safety of eculizumab in patients with NMO [ Time Frame: 15 months ] [ Designated as safety issue: Yes ]
- Improvement of quality of life [ Time Frame: 15 months ] [ Designated as safety issue: No ]
- Improvement in visual function [ Time Frame: 15 months ] [ Designated as safety issue: No ]
- Improvement in walking [ Time Frame: 15 months ] [ Designated as safety issue: No ]
- Pharmacokinetics of the drug in blood [ Time Frame: 15 months ] [ Designated as safety issue: No ]
- Pharmacokinetics of drug in CSF [ Time Frame: 15 months ] [ Designated as safety issue: No ]
|Study Start Date:||April 2009|
|Study Completion Date:||December 2011|
|Primary Completion Date:||December 2011 (Final data collection date for primary outcome measure)|
The patient will receive eculizumab at a dose of 600mg intravenously (an infusion given into the vein) each week for 4 weeks, then 900mg intravenously at the fifth week, then 900mg every 2 weeks for 48 weeks.
The first infusion will be given at Mayo Clinic site; subsequent infusions will be administered in your home by a company which will send a nurse to the patient's home to administer the infusion (Coram Home infusion services). This will be coordinated by either a Study physician or coordinator.
Other Name: Soliris
It has been shown in some scientific studies that the the antibody marker specific for neuromyelitis optica (NMO), known as NMO-IgG,causes inflammation in brain tissues by activating a substance called complement. Complement can greatly increase the immune attack in the optic nerves (causing optic neuritis [ON]), spinal cords (causing transverse myelitis [TM]) and brains of patients with NMO. Eculizumab has already been shown to be effective in a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). Attacks of PNH are also mediated through complement. Therefore, the investigators of this study are investigating whether by 'turning off' complement in NMO, further attacks of NMO can be prevented.
The primary (most important) objectives of this study are to determine:
Whether Eculizumab 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 eculizumab treatment. For patients with more than 2 year disease duration, the average number of attacks in the preceding 2 years will be calculated. For patients with less than 2 years disease duration the number of attacks in the preceding year will be used.
The safety profile of eculizumab in patients with NMO
The secondary objectives are to determine:
Whether eculizumab 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.
How the drug behaves in the patient's blood (called pharmacodynamics and pharmacokinetics)
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.
|United States, Arizona|
|Scottsdale, Arizona, United States, 85259|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Sean J. Pittock, M.D.||Mayo Clinic|