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A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)

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ClinicalTrials.gov Identifier: NCT01892345
Recruitment Status : Terminated
First Posted : July 4, 2013
Results First Posted : June 26, 2019
Last Update Posted : June 26, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Interventions Drug: Eculizumab
Drug: Placebo
Enrollment 143
Recruitment Details  
Pre-assignment Details Main inclusion criteria were: participants aged ≥ 18 years old with NMO/NMOSD, AQP4 antibody-positive, historical relapse of at least 2 in last 12 months or 3 in last 24 months with at least 1 in 12 months prior to screening, Expanded Disability Status Scale score ≤ 7. If receiving immunosuppressive therapies, must be on a stable maintenance dose.
Arm/Group Title Eculizumab Placebo
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Induction Period: Participants received eculizumab (900 milligrams [mg]) via intravenous (IV) infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Period Title: Overall Study
Started 96 47
Received At Least 1 Dose Of Study Drug [1] 96 47
Completed 80 44
Not Completed 16 3
Reason Not Completed
Adverse Event             0             2
Death             1             0
Lost to Follow-up             3             0
Withdrawal by Subject             12             1
[1]
Full Analysis Set (FAS)
Arm/Group Title Eculizumab Placebo Total
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Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Total of all reporting groups
Overall Number of Baseline Participants 96 47 143
Hide Baseline Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 96 participants 47 participants 143 participants
43.9  (13.32) 45.0  (13.29) 44.3  (13.27)
[1]
Measure Description: Age at first dose (years)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 96 participants 47 participants 143 participants
Female
88
  91.7%
42
  89.4%
130
  90.9%
Male
8
   8.3%
5
  10.6%
13
   9.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 96 participants 47 participants 143 participants
Hispanic or Latino
13
  13.5%
3
   6.4%
16
  11.2%
Not Hispanic or Latino
78
  81.3%
41
  87.2%
119
  83.2%
Unknown or Not Reported
5
   5.2%
3
   6.4%
8
   5.6%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 96 participants 47 participants 143 participants
Asian
37
  38.5%
15
  31.9%
52
  36.4%
Black or African American
9
   9.4%
8
  17.0%
17
  11.9%
White
46
  47.9%
24
  51.1%
70
  49.0%
Other or Unknown
4
   4.2%
0
   0.0%
4
   2.8%
Overall Stratification Groupings (4 strata) at Randomization   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 96 participants 47 participants 143 participants
Low EDSS (≤ 2.0)
11
  11.5%
5
  10.6%
16
  11.2%
High EDSS (≥ 2.5 to ≤ 7) and Treatment Naive
12
  12.5%
5
  10.6%
17
  11.9%
High EDSS (≥ 2.5 to ≤ 7): Same IST
44
  45.8%
22
  46.8%
66
  46.2%
High EDSS (≥ 2.5 to ≤ 7): Change in IST
29
  30.2%
15
  31.9%
44
  30.8%
[1]
Measure Description:

EDSS = Expanded Disability Status Scale; IST = Immunosuppressive Therapy

High EDSS (≥ 2.5 to ≤ 7): Same IST = High EDSS (≥ 2.5 to ≤ 7) and Continuing on the Same IST(s) since last relapse.

High EDSS (≥ 2.5 to ≤ 7): Change in IST = High EDSS (≥ 2.5 to ≤ 7) and Changes in IST(s) since last relapse.

1.Primary Outcome
Title Participants With An Adjudicated On-trial Relapse
Hide Description 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. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment, received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
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Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Measure Type: Count of Participants
Unit of Measure: Participants
3
   3.1%
20
  42.6%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment Effect
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Stratified Log-Rank Test
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.058
Confidence Interval (2-Sided) 95%
0.017 to 0.197
Estimation Comments HR based on a stratified Cox proportional hazards model. Confidence interval = Wald confidence interval. HR for eculizumab compared with placebo represented a 94.2% reduction in the risk of relapse, 95% Wald confidence interval (80.3%, 98.3%).
2.Secondary Outcome
Title Adjudicated On-trial Annualized Relapse Rate (ARR)
Hide Description The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: relapses/years on study
0.016
(0.005 to 0.050)
0.350
(0.199 to 0.616)
3.Secondary Outcome
Title Change From Baseline In EDSS At End Of Study
Hide Description Disease-related disability was measured by the EDSS. 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.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.18  (0.814) 0.12  (0.945)
4.Secondary Outcome
Title Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
Hide Description 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 disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.2  (0.72) 0.1  (0.75)
5.Secondary Outcome
Title Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
Hide Description 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 ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.4  (1.08) 0.5  (1.61)
6.Secondary Outcome
Title Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
Hide Description The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual “thermometer” with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
5.4  (18.53) 0.6  (16.39)
7.Secondary Outcome
Title Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
Hide Description The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status.
Time Frame Baseline, Up To 211 Weeks (End of Study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Overall Number of Participants Analyzed 96 47
Mean (Standard Deviation)
Unit of Measure: units on a scale
0.05  (0.179) -0.04  (0.212)
Time Frame From Day 1 to End of Study (211 Weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description

Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks followed by eculizumab 1200 mg for the fifth dose (Week 4).

Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

Induction Period: Participants received matching placebo (900 mg) via IV infusion once a week (every 7 ± 2 days) for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose (Week 4).

Maintenance Period: Participants received matching placebo (1200 mg) via IV infusion every 2 weeks (every 14 ± 2 days) from the sixth dose (Week 6) onwards.

All-Cause Mortality
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/96 (1.04%)   0/47 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   30/96 (31.25%)   26/47 (55.32%) 
Blood and lymphatic system disorders     
Leukocytosis  1  1/96 (1.04%)  0/47 (0.00%) 
Pancytopenia  1  0/96 (0.00%)  1/47 (2.13%) 
Thrombocytopenia  1  0/96 (0.00%)  1/47 (2.13%) 
Cardiac disorders     
Atrial fibrillation  1  1/96 (1.04%)  0/47 (0.00%) 
Cardiac failure congestive  1  1/96 (1.04%)  0/47 (0.00%) 
Myocardial ischaemia  1  0/96 (0.00%)  1/47 (2.13%) 
Ear and labyrinth disorders     
Vertigo  1  0/96 (0.00%)  1/47 (2.13%) 
Eye disorders     
Cataract  1  1/96 (1.04%)  0/47 (0.00%) 
Conjunctival haemorrhage  1  1/96 (1.04%)  0/47 (0.00%) 
Visual impairment  1  1/96 (1.04%)  0/47 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  0/96 (0.00%)  1/47 (2.13%) 
Abdominal pain upper  1  1/96 (1.04%)  0/47 (0.00%) 
Nausea  1  1/96 (1.04%)  0/47 (0.00%) 
Pancreatitis  1  0/96 (0.00%)  1/47 (2.13%) 
Umbilical hernia  1  1/96 (1.04%)  0/47 (0.00%) 
Vomiting  1  1/96 (1.04%)  0/47 (0.00%) 
General disorders     
Pain  1  1/96 (1.04%)  0/47 (0.00%) 
Pyrexia  1  1/96 (1.04%)  0/47 (0.00%) 
Hepatobiliary disorders     
Cholecystitis acute  1  1/96 (1.04%)  1/47 (2.13%) 
Infections and infestations     
Appendicitis  1  1/96 (1.04%)  0/47 (0.00%) 
Bartholin's abscess  1  1/96 (1.04%)  0/47 (0.00%) 
Bronchitis  1  1/96 (1.04%)  1/47 (2.13%) 
Cellulitis  1  2/96 (2.08%)  0/47 (0.00%) 
Gallbladder empyema  1  1/96 (1.04%)  0/47 (0.00%) 
Gastroenteritis viral  1  0/96 (0.00%)  1/47 (2.13%) 
Herpes zoster  1  0/96 (0.00%)  1/47 (2.13%) 
Infectious pleural effusion  1  1/96 (1.04%)  0/47 (0.00%) 
Influenza  1  1/96 (1.04%)  1/47 (2.13%) 
Pneumococcal infection  1  0/96 (0.00%)  1/47 (2.13%) 
Pneumonia  1  3/96 (3.13%)  1/47 (2.13%) 
Renal abscess  1  1/96 (1.04%)  0/47 (0.00%) 
Sepsis  1  2/96 (2.08%)  0/47 (0.00%) 
Urinary tract infection  1  2/96 (2.08%)  0/47 (0.00%) 
Viral upper respiratory tract infection  1  0/96 (0.00%)  1/47 (2.13%) 
Injury, poisoning and procedural complications     
Cervical vertebral fracture  1  1/96 (1.04%)  0/47 (0.00%) 
Contusion  1  1/96 (1.04%)  0/47 (0.00%) 
Fall  1  1/96 (1.04%)  0/47 (0.00%) 
Hip fracture  1  1/96 (1.04%)  0/47 (0.00%) 
Pubis fracture  1  1/96 (1.04%)  0/47 (0.00%) 
Rib fracture  1  0/96 (0.00%)  1/47 (2.13%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  1/96 (1.04%)  0/47 (0.00%) 
Muscular weakness  1  0/96 (0.00%)  1/47 (2.13%) 
Pain in extremity  1  1/96 (1.04%)  0/47 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Adenocarcinoma  1  0/96 (0.00%)  1/47 (2.13%) 
Nervous system disorders     
Myelitis transverse  1  0/96 (0.00%)  1/47 (2.13%) 
Neuromyelitis optica spectrum disorder  1  7/96 (7.29%)  16/47 (34.04%) 
Paraesthesia  1  0/96 (0.00%)  1/47 (2.13%) 
Somnolence  1  0/96 (0.00%)  1/47 (2.13%) 
Syncope  1  0/96 (0.00%)  1/47 (2.13%) 
Psychiatric disorders     
Confusional state  1  0/96 (0.00%)  1/47 (2.13%) 
Panic attack  1  0/96 (0.00%)  1/47 (2.13%) 
Renal and urinary disorders     
Haematuria  1  1/96 (1.04%)  0/47 (0.00%) 
Urinary retention  1  1/96 (1.04%)  0/47 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  1/96 (1.04%)  0/47 (0.00%) 
Dyspnoea  1  1/96 (1.04%)  0/47 (0.00%) 
Dyspnoea exertional  1  1/96 (1.04%)  0/47 (0.00%) 
Pleurisy  1  0/96 (0.00%)  1/47 (2.13%) 
Pulmonary embolism  1  0/96 (0.00%)  1/47 (2.13%) 
Respiratory disorder  1  0/96 (0.00%)  1/47 (2.13%) 
Respiratory failure  1  1/96 (1.04%)  0/47 (0.00%) 
Surgical and medical procedures     
Catheterisation venous  1  1/96 (1.04%)  0/47 (0.00%) 
Vascular disorders     
Orthostatic hypotension  1  0/96 (0.00%)  1/47 (2.13%) 
Superior mesenteric artery syndrome  1  1/96 (1.04%)  0/47 (0.00%) 
Venous occlusion  1  1/96 (1.04%)  0/47 (0.00%) 
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   86/96 (89.58%)   43/47 (91.49%) 
Blood and lymphatic system disorders     
Leukopenia  1  5/96 (5.21%)  1/47 (2.13%) 
Lymphopenia  1  5/96 (5.21%)  0/47 (0.00%) 
Eye disorders     
Cataract  1  6/96 (6.25%)  2/47 (4.26%) 
Gastrointestinal disorders     
Abdominal pain upper  1  5/96 (5.21%)  3/47 (6.38%) 
Constipation  1  9/96 (9.38%)  3/47 (6.38%) 
Diarrhoea  1  15/96 (15.63%)  7/47 (14.89%) 
Dyspepsia  1  6/96 (6.25%)  4/47 (8.51%) 
Gastrooesophageal reflux disease  1  5/96 (5.21%)  3/47 (6.38%) 
Nausea  1  16/96 (16.67%)  12/47 (25.53%) 
Vomiting  1  9/96 (9.38%)  8/47 (17.02%) 
General disorders     
Asthenia  1  5/96 (5.21%)  1/47 (2.13%) 
Chest discomfort  1  2/96 (2.08%)  3/47 (6.38%) 
Fatigue  1  7/96 (7.29%)  5/47 (10.64%) 
Oedema peripheral  1  4/96 (4.17%)  3/47 (6.38%) 
Pain  1  4/96 (4.17%)  4/47 (8.51%) 
Pyrexia  1  6/96 (6.25%)  4/47 (8.51%) 
Infections and infestations     
Bronchitis  1  9/96 (9.38%)  2/47 (4.26%) 
Conjunctivitis  1  9/96 (9.38%)  4/47 (8.51%) 
Cystitis  1  8/96 (8.33%)  1/47 (2.13%) 
Hordeolum  1  7/96 (7.29%)  0/47 (0.00%) 
Influenza  1  11/96 (11.46%)  1/47 (2.13%) 
Nasopharyngitis  1  20/96 (20.83%)  9/47 (19.15%) 
Pharyngitis  1  10/96 (10.42%)  3/47 (6.38%) 
Pneumonia  1  0/96 (0.00%)  3/47 (6.38%) 
Sinusitis  1  6/96 (6.25%)  0/47 (0.00%) 
Upper respiratory tract infection  1  28/96 (29.17%)  6/47 (12.77%) 
Urinary tract infection  1  11/96 (11.46%)  10/47 (21.28%) 
Injury, poisoning and procedural complications     
Contusion  1  9/96 (9.38%)  2/47 (4.26%) 
Fall  1  3/96 (3.13%)  4/47 (8.51%) 
Investigations     
Weight decreased  1  1/96 (1.04%)  3/47 (6.38%) 
Metabolism and nutrition disorders     
Decreased appetite  1  5/96 (5.21%)  1/47 (2.13%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  11/96 (11.46%)  5/47 (10.64%) 
Back pain  1  14/96 (14.58%)  6/47 (12.77%) 
Muscle spasms  1  5/96 (5.21%)  2/47 (4.26%) 
Musculoskeletal pain  1  6/96 (6.25%)  0/47 (0.00%) 
Myalgia  1  6/96 (6.25%)  3/47 (6.38%) 
Pain in extremity  1  10/96 (10.42%)  10/47 (21.28%) 
Nervous system disorders     
Dizziness  1  14/96 (14.58%)  6/47 (12.77%) 
Headache  1  22/96 (22.92%)  11/47 (23.40%) 
Hypoaesthesia  1  2/96 (2.08%)  4/47 (8.51%) 
Paraesthesia  1  8/96 (8.33%)  3/47 (6.38%) 
Psychiatric disorders     
Depression  1  1/96 (1.04%)  4/47 (8.51%) 
Insomnia  1  6/96 (6.25%)  4/47 (8.51%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  10/96 (10.42%)  7/47 (14.89%) 
Nasal congestion  1  2/96 (2.08%)  3/47 (6.38%) 
Oropharyngeal pain  1  7/96 (7.29%)  2/47 (4.26%) 
Rhinitis allergic  1  3/96 (3.13%)  3/47 (6.38%) 
Skin and subcutaneous tissue disorders     
Alopecia  1  5/96 (5.21%)  2/47 (4.26%) 
Pruritus  1  3/96 (3.13%)  4/47 (8.51%) 
Rash  1  4/96 (4.17%)  4/47 (8.51%) 
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
After rigorous review of blinded study data, the Sponsor terminated the study at 23 adjudicated events, not the protocol-specified 24. This was not driven by safety or efficacy concerns, but rather by uncertainty in estimating final event occurrence.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Publication rights are tied to the completion of the multi-center publication.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals, Inc.
Organization: Alexion Pharmaceuticals, Inc.
Phone: 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01892345     History of Changes
Other Study ID Numbers: ECU-NMO-301
First Submitted: June 20, 2013
First Posted: July 4, 2013
Results First Submitted: June 7, 2019
Results First Posted: June 26, 2019
Last Update Posted: June 26, 2019