An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica

Treatment tested

• Eculizumab

  Complement C5 inhibitor

Other measures (8)

Inclusion Criteria: 1. Diagnosis of NMO, as defined by 2006 criteria OR NMO seropositive spectrum disorder (Recurrent ON or longitudinally extensive transverse myelitis (LETM)). All patients must be NMO-IgG seropositive. 2. Clinical evidence of at least 2 relapses in last 6 months or 3 relapses in the last 12 months (with at least 1 relapse occurring in the preceding 6 months). 3. Age ≥18 years 4. Corrected visual acuity 20/100 or better in at least one eye. If fails item # 4 then entry allowed but only if last attack was myelitis and only attacks of myelitis are considered as outcome measurement. 5. Ambulatory (with or without walker). If fails item # 5 then entry allowed but only if last attack was ON and only attacks of ON are considered as outcome measurement. 6. Provision of written informed consent (see attached) to participate in the study. 7. N. meningitidis vaccination at least 14 days prior to receiving the first eculizumab infusion. If patient in midst of an acute relapse, then relapse will be treated with standard therapy and vaccination given only after a minimum of 4 weeks post attack onset. Exclusion Criteria: Candidates will be excluded from study entry if any of the following criteria are met at the time of randomization: 1. Progressive neurological deterioration unrelated to relapses of ON or myelitis. 2. Pregnant, breastfeeding, or intending to conceive during the course of the study 3. Patients will not participate in any other clinical therapeutic study or will not have participated in any other experimental treatment study within 30 days of screening 4. Patients with a history of splenectomy, because of a potential increased risk of developing meningococcal infection.

The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.