Table 2.

Current randomized controlled trials for prevention of attacks in neuromyelitis optica spectrum disorders (NMOSD)

Study number	Compound (company)	Target structure	Trial design	Regimen	Patients	Estimated recruitment (n)	Study duration	End point, comments
NCT01892345	Eculizumab (Alexion)	C5	Phase III, placebo-controlled, double blind, add-on to immunosuppressive therapy OR as monotherapy	900 mg IV every week for 4 weeks, followed by 1200 mg IV every 2 weeks	AQP4-pos. NMO/NMOSD, age ≥ 18 years, EDSS ≤ 7.0	132	104 weeks	Time to first attack, randomization 2:1, study followed by open-label extension, completion of double-blind part of study estimated 2016
NCT02028884	SA237 (Chugai)	IL-6R	Phase III, placebo-controlled, double blind, add-on to oral corticosteroids or AZA, or MMF (EU, Japan, Taiwan)	120 mg SC at weeks 0, 2, and 4; thereafter, every 4 weeks	NMO or AQP4-pos. NMOSD, age 12–74 years	70	Up to 30 months	Time to first attack, randomization 1:1, double blind followed by open-label extension, completion of double-blind part of study estimated 2017
NCT02073279	SA237 (Chugai)	IL-6R	Phase III, placebo-controlled, double blind, monotherapy (North America)	120 mg SC at weeks 0, 2, and 4; thereafter, every 4 weeks	NMO or AQP4-pos. NMOSD, age 18–74 years	70	Up to 30 months	Time to first attack, randomization 1:2, double blind followed by open-label extension, completion of double-blind part of study estimated 2017
NCT02200770	MEDI-551 (MedImmune)	CD19	Phase II/III placebo-controlled, double blind, monotherapy	300 mg IV on days 1 and 15, and then every 6 months	NMO or AQP4-pos. NMOSD, age ≥ 18 years, EDSS ≤ 7.5	212	3 years	Time to first attack, randomization 3:1, maximum 197 days placebo, followed by open-label period, completion estimated 2019

IV = intravenously; AQP4 = aquaporin-4-antibody; EDSS = expanded disability status scale; IL-6R = interleukin-6 receptor; AZA = azathioprine; MMF = mycophenolate mofetil; EU = European Union; SC = subcutaneously