Trial Outcomes & Findings for A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study) (NCT NCT01892345)
NCT ID: NCT01892345
Last Updated: 2019-06-26
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
143 participants
Primary outcome timeframe
Baseline, Up To 211 Weeks (End of Study)
Results posted on
2019-06-26
Participant Flow
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.
Participant milestones
| Measure |
Eculizumab
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.
|
Placebo
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 Study
STARTED
|
96
|
47
|
|
Overall Study
Received At Least 1 Dose Of Study Drug
|
96
|
47
|
|
Overall Study
COMPLETED
|
80
|
44
|
|
Overall Study
NOT COMPLETED
|
16
|
3
|
Reasons for withdrawal
| Measure |
Eculizumab
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.
|
Placebo
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 Study
Adverse Event
|
0
|
2
|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
3
|
0
|
|
Overall Study
Withdrawal by Subject
|
12
|
1
|
Baseline Characteristics
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
Baseline characteristics by cohort
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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
n=143 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
43.9 years
STANDARD_DEVIATION 13.32 • n=5 Participants
|
45.0 years
STANDARD_DEVIATION 13.29 • n=7 Participants
|
44.3 years
STANDARD_DEVIATION 13.27 • n=5 Participants
|
|
Sex: Female, Male
Female
|
88 Participants
n=5 Participants
|
42 Participants
n=7 Participants
|
130 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
13 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
78 Participants
n=5 Participants
|
41 Participants
n=7 Participants
|
119 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
37 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
52 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
46 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
70 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other or Unknown
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Overall Stratification Groupings (4 strata) at Randomization
Low EDSS (≤ 2.0)
|
11 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Overall Stratification Groupings (4 strata) at Randomization
High EDSS (≥ 2.5 to ≤ 7) and Treatment Naive
|
12 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Overall Stratification Groupings (4 strata) at Randomization
High EDSS (≥ 2.5 to ≤ 7): Same IST
|
44 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
66 Participants
n=5 Participants
|
|
Overall Stratification Groupings (4 strata) at Randomization
High EDSS (≥ 2.5 to ≤ 7): Change in IST
|
29 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment, received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Participants With An Adjudicated On-trial Relapse
|
3 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Adjudicated On-trial Annualized Relapse Rate (ARR)
|
0.016 relapses/years on study
Interval 0.005 to 0.05
|
0.350 relapses/years on study
Interval 0.199 to 0.616
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Change From Baseline In EDSS At End Of Study
|
-0.18 units on a scale
Standard Deviation 0.814
|
0.12 units on a scale
Standard Deviation 0.945
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
|
-0.2 units on a scale
Standard Deviation 0.72
|
0.1 units on a scale
Standard Deviation 0.75
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
|
-0.4 units on a scale
Standard Deviation 1.08
|
0.5 units on a scale
Standard Deviation 1.61
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
|
5.4 units on a scale
Standard Deviation 18.53
|
0.6 units on a scale
Standard Deviation 16.39
|
SECONDARY outcome
Timeframe: Baseline, Up To 211 Weeks (End of Study)Population: Full Analysis Set (FAS): all participants who were randomized to treatment and who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
Eculizumab
n=96 Participants
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.
|
Placebo
n=47 Participants
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.
|
|---|---|---|
|
Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
|
0.05 units on a scale
Standard Deviation 0.179
|
-0.04 units on a scale
Standard Deviation 0.212
|
Adverse Events
Eculizumab
Serious events: 30 serious events
Other events: 86 other events
Deaths: 1 deaths
Placebo
Serious events: 26 serious events
Other events: 43 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eculizumab
n=96 participants at risk
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.
|
Placebo
n=47 participants at risk
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.
|
|---|---|---|
|
Blood and lymphatic system disorders
Leukocytosis
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Cardiac disorders
Atrial fibrillation
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Cardiac disorders
Cardiac failure congestive
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Cardiac disorders
Myocardial ischaemia
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Eye disorders
Cataract
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Eye disorders
Conjunctival haemorrhage
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Eye disorders
Visual impairment
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Nausea
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Pancreatitis
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Umbilical hernia
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Vomiting
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Pain
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Pyrexia
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Hepatobiliary disorders
Cholecystitis acute
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Appendicitis
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Bartholin's abscess
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Bronchitis
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Cellulitis
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Gallbladder empyema
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Infectious pleural effusion
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Influenza
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Pneumococcal infection
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Pneumonia
|
3.1%
3/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Renal abscess
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Sepsis
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Urinary tract infection
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Cervical vertebral fracture
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Contusion
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Fall
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Hip fracture
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Pubis fracture
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Myelitis transverse
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Neuromyelitis optica spectrum disorder
|
7.3%
7/96 • From Day 1 to End of Study (211 Weeks)
|
34.0%
16/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Somnolence
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Syncope
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Psychiatric disorders
Panic attack
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Renal and urinary disorders
Haematuria
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Pleurisy
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Renal and urinary disorders
Urinary retention
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Surgical and medical procedures
Catheterisation venous
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Vascular disorders
Orthostatic hypotension
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Vascular disorders
Superior mesenteric artery syndrome
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Vascular disorders
Venous occlusion
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
Other adverse events
| Measure |
Eculizumab
n=96 participants at risk
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.
|
Placebo
n=47 participants at risk
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.
|
|---|---|---|
|
Infections and infestations
Hordeolum
|
7.3%
7/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Influenza
|
11.5%
11/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Nasopharyngitis
|
20.8%
20/96 • From Day 1 to End of Study (211 Weeks)
|
19.1%
9/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Pharyngitis
|
10.4%
10/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Pneumonia
|
0.00%
0/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Blood and lymphatic system disorders
Leukopenia
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Blood and lymphatic system disorders
Lymphopenia
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Eye disorders
Cataract
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Constipation
|
9.4%
9/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Diarrhoea
|
15.6%
15/96 • From Day 1 to End of Study (211 Weeks)
|
14.9%
7/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Dyspepsia
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Nausea
|
16.7%
16/96 • From Day 1 to End of Study (211 Weeks)
|
25.5%
12/47 • From Day 1 to End of Study (211 Weeks)
|
|
Gastrointestinal disorders
Vomiting
|
9.4%
9/96 • From Day 1 to End of Study (211 Weeks)
|
17.0%
8/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Asthenia
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Chest discomfort
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Fatigue
|
7.3%
7/96 • From Day 1 to End of Study (211 Weeks)
|
10.6%
5/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Oedema peripheral
|
4.2%
4/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Pain
|
4.2%
4/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
General disorders
Pyrexia
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Bronchitis
|
9.4%
9/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Conjunctivitis
|
9.4%
9/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Cystitis
|
8.3%
8/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Sinusitis
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Upper respiratory tract infection
|
29.2%
28/96 • From Day 1 to End of Study (211 Weeks)
|
12.8%
6/47 • From Day 1 to End of Study (211 Weeks)
|
|
Infections and infestations
Urinary tract infection
|
11.5%
11/96 • From Day 1 to End of Study (211 Weeks)
|
21.3%
10/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Contusion
|
9.4%
9/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Injury, poisoning and procedural complications
Fall
|
3.1%
3/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Investigations
Weight decreased
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
2.1%
1/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
11.5%
11/96 • From Day 1 to End of Study (211 Weeks)
|
10.6%
5/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.6%
14/96 • From Day 1 to End of Study (211 Weeks)
|
12.8%
6/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
0.00%
0/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.4%
10/96 • From Day 1 to End of Study (211 Weeks)
|
21.3%
10/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Dizziness
|
14.6%
14/96 • From Day 1 to End of Study (211 Weeks)
|
12.8%
6/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Headache
|
22.9%
22/96 • From Day 1 to End of Study (211 Weeks)
|
23.4%
11/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Hypoaesthesia
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Nervous system disorders
Paraesthesia
|
8.3%
8/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Psychiatric disorders
Depression
|
1.0%
1/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Psychiatric disorders
Insomnia
|
6.2%
6/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.4%
10/96 • From Day 1 to End of Study (211 Weeks)
|
14.9%
7/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
2.1%
2/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.3%
7/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
3.1%
3/96 • From Day 1 to End of Study (211 Weeks)
|
6.4%
3/47 • From Day 1 to End of Study (211 Weeks)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
5.2%
5/96 • From Day 1 to End of Study (211 Weeks)
|
4.3%
2/47 • From Day 1 to End of Study (211 Weeks)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
3.1%
3/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.2%
4/96 • From Day 1 to End of Study (211 Weeks)
|
8.5%
4/47 • From Day 1 to End of Study (211 Weeks)
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: 855-752-2356
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Publication rights are tied to the completion of the multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER