18 and older, any sex, with Neuromyelitis Optica or Neuromyelitis Optica Spectrum Disorder. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse EventsPrimary· Baseline up to end of study (up to 6.5 years)
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of exi
TEAEs
Group
Value
95% CI
Placebo/Eculizumab
41
Eculizumab/Eculizumab
70
SAEs
Group
Value
95% CI
Placebo/Eculizumab
14
Eculizumab/Eculizumab
26
Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) AbnormalityPrimary· Baseline up to end of study (up to 6.5 years)
The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparato
Suicidal Ideation
Group
Value
95% CI
Placebo/Eculizumab
4
Eculizumab/Eculizumab
5
Suicidal Behavior
Group
Value
95% CI
Placebo/Eculizumab
0
Eculizumab/Eculizumab
1
Suicidal Ideation or Behavior
Group
Value
95% CI
Placebo/Eculizumab
4
Eculizumab/Eculizumab
5
Number of Participants With An On-trial Relapse as Determined by The Treating PhysicianPrimary· Baseline up to end of study (up to 6.5 years)
An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician.
Group
Value
95% CI
Placebo/Eculizumab
5
Eculizumab/Eculizumab
8
On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating PhysicianPrimary· Baseline up to end of study (up to 6.5 years)
The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.
Group
Value
95% CI
Placebo/Eculizumab
0.128
± 0.4576
Eculizumab/Eculizumab
0.061
± 0.2186
Change From Baseline in Expanded Disability Status Scale (EDSS) ScoreSecondary· Baseline, Weeks 52, 104 and 156
Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-
Baseline
Group
Value
95% CI
Placebo/Eculizumab
4.34
± 1.879
Eculizumab/Eculizumab
3.97
± 1.736
Change from Baseline at Week 52
Group
Value
95% CI
Placebo/Eculizumab
-0.24
± 0.721
Eculizumab/Eculizumab
0.01
± 0.571
Change from Baseline at Week 104
Group
Value
95% CI
Placebo/Eculizumab
-0.39
± 0.830
Eculizumab/Eculizumab
-0.11
± 0.536
Change from Baseline at Week 156
Group
Value
95% CI
Placebo/Eculizumab
-0.38
± 1.003
Eculizumab/Eculizumab
-0.38
± 1.057
Change From Baseline in Modified Rankin Scale (mRS) ScoreSecondary· Baseline, Weeks 52, 104 and 156
Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Baseline
Group
Value
95% CI
Placebo/Eculizumab
2.39
± 1.358
Eculizumab/Eculizumab
1.88
± 1.269
Change from Baseline at Week 52
Group
Value
95% CI
Placebo/Eculizumab
-0.27
± 0.932
Eculizumab/Eculizumab
-0.04
± 0.458
Change from Baseline at Week 104
Group
Value
95% CI
Placebo/Eculizumab
-0.41
± 1.182
Eculizumab/Eculizumab
-0.14
± 0.543
Change from Baseline at Week 156
Group
Value
95% CI
Placebo/Eculizumab
-0.62
± 1.446
Eculizumab/Eculizumab
-0.31
± 0.602
Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory FunctionSecondary· Baseline, Weeks 52, 104 and 156
The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Baseline
Group
Value
95% CI
Placebo/Eculizumab
2.83
± 2.123
Eculizumab/Eculizumab
2.35
± 2.257
Change from Baseline at Week 52
Group
Value
95% CI
Placebo/Eculizumab
-0.44
± 1.132
Eculizumab/Eculizumab
0.08
± 0.816
Change from Baseline at Week 104
Group
Value
95% CI
Placebo/Eculizumab
-0.57
± 1.777
Eculizumab/Eculizumab
0.07
± 1.033
Change from Baseline at Week 156
Group
Value
95% CI
Placebo/Eculizumab
-1.08
± 1.706
Eculizumab/Eculizumab
0.07
± 1.269
Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status ScoreSecondary· Baseline, Weeks 52, 104 and 156
The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Baseline
Group
Value
95% CI
Placebo/Eculizumab
62.00
± 22.012
Eculizumab/Eculizumab
72.27
± 20.941
Change from Baseline at Week 52
Group
Value
95% CI
Placebo/Eculizumab
2.22
± 13.294
Eculizumab/Eculizumab
-0.78
± 12.388
Change from Baseline at Week 104
Group
Value
95% CI
Placebo/Eculizumab
0.05
± 18.867
Eculizumab/Eculizumab
1.28
± 11.295
Change from Baseline at Week 156
Group
Value
95% CI
Placebo/Eculizumab
11.00
± 19.374
Eculizumab/Eculizumab
-4.13
± 18.421
Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual FunctionSecondary· Baseline, Weeks 52, 104 and 156
The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302.
Baseline
Group
Value
95% CI
Placebo/Eculizumab
3.75
± 2.030
Eculizumab/Eculizumab
3.60
± 2.031
Change from Baseline at Week 52
Group
Value
95% CI
Placebo/Eculizumab
-0.08
± 0.392
Eculizumab/Eculizumab
-0.06
± 0.569
Change from Baseline at Week 104
Group
Value
95% CI
Placebo/Eculizumab
-0.13
± 0.352
Eculizumab/Eculizumab
-0.10
± 0.651
Change from Baseline at Week 156
Group
Value
95% CI
Placebo/Eculizumab
0.00
± 0.000
Eculizumab/Eculizumab
-0.29
± 0.994
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline up to end of study (up to 6.5 years).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
Last refreshed: 23 August 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02003144.