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A Clinical Trial of Omalizumab in Participants With Chronic Rhinosinusitus With Nasal Polyps (POLYP 2)

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ClinicalTrials.gov Identifier: NCT03280537
Recruitment Status : Completed
First Posted : September 12, 2017
Results First Posted : March 23, 2020
Last Update Posted : March 23, 2020
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Nasal Polyps
Chronic Rhinosinusitis
Interventions Drug: Omalizumab
Drug: Placebo
Enrollment 127
Recruitment Details  
Pre-assignment Details At the first visit of the 5-week screening/run-in period, participants were asked to standardize their nasal corticosteroids to a regimen of mometasone, 200 micrograms twice a day (BID). If intolerant to a BID regimen, then they remained on a stable dosage of mometasone once a day (QD) during the run-in period and throughout the treatment period.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Period Title: Overall Study
Started 65 62
Completed 63 58
Not Completed 2 4
Reason Not Completed
Withdrawal by Subject             2             4
Arm/Group Title Placebo Omalizumab Total
Hide Arm/Group Description Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. Total of all reporting groups
Overall Number of Baseline Participants 65 62 127
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 65 participants 62 participants 127 participants
51.0  (12.0) 49.0  (11.9) 50.1  (11.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
Female
21
  32.3%
23
  37.1%
44
  34.6%
Male
44
  67.7%
39
  62.9%
83
  65.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
Hispanic or Latino
3
   4.6%
5
   8.1%
8
   6.3%
Not Hispanic or Latino
61
  93.8%
56
  90.3%
117
  92.1%
Unknown or Not Reported
1
   1.5%
1
   1.6%
2
   1.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
65
 100.0%
61
  98.4%
126
  99.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   1.6%
1
   0.8%
Geographic Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
North America
14
  21.5%
12
  19.4%
26
  20.5%
ex-North America
51
  78.5%
50
  80.6%
101
  79.5%
Participants with Asthma Comorbidity and Aspirin Sensitivity   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
Asthmatic and Aspirin Sensitive
18
  27.7%
21
  33.9%
39
  30.7%
Asthmatic and Not Aspirin Sensitive
21
  32.3%
17
  27.4%
38
  29.9%
Not Asthmatic
26
  40.0%
24
  38.7%
50
  39.4%
[1]
Measure Description: Asthma comorbidity was defined as asthma history at screening and having used medication for asthma or received a prescription for any asthma medication in the last 12 months prior to screening.
Average Daily Nasal Congestion Score at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
2.3  (0.6) 2.3  (0.7) 2.3  (0.7)
[1]
Measure Description: The Nasal Congestion Score (NCS) was assessed daily by the participant via an electronic diary as the response to the following question: Is your nose blocked? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. Baseline was defined as the average of the daily values recorded during the 7-day interval ending on the latest day prior to randomization such that the prior 7-day interval includes a recorded value on at least 4 of the 7 days of that interval.
Nasal Polyp Score (NPS) at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
6.1  (0.9) 6.4  (0.9) 6.3  (0.9)
[1]
Measure Description: Total NPS ranges from 0 to 8 (sum of 0-4 for left and right nasal passage scores per the following criteria), with a lower score indicating smaller-sized nasal polyps: 0 = No polyps; 1 = Small polyps in middle meatus not reaching below inferior border of the middle turbinate; 2 = Polyps reaching below lower border of the middle turbinate; 3 = Large polyps reaching lower border of the inferior turbinate or polyps medial to the middle turbinate; and 4 = Large polyps causing complete obstruction of the inferior nasal cavity. Baseline was the last assessment on or before the date of randomization.
Average Daily Total Nasal Symptom Score (TNSS) at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
8.7  (2.3) 8.4  (2.6) 8.6  (2.4)
[1]
Measure Description: The Total Nasal Symptom Score (TNSS) was defined as the sum of the four individual scores for Nasal Congestion Score, Anterior Rhinorrhea Score, Posterior Rhinorrhea Score, and Sense of Smell Score, ranging from 0 (no symptoms) to 12 (most severe symptoms), assessed daily by the participant via an electronic diary. Baseline was defined as the average of the daily values recorded during the 7-day interval ending on the latest day prior to randomization such that the prior 7-day interval includes a recorded value on at least 4 of the 7 days of that interval.
Average Daily Sense of Smell Score at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
2.8  (0.6) 2.6  (0.8) 2.7  (0.7)
[1]
Measure Description: The Sense of Smell Score was assessed daily by the participant via an electronic diary as the response to the following question: Is your sense of smell reduced? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. Baseline was defined as the average of the daily values recorded during the 7-day interval ending on the latest day prior to randomization such that the prior 7-day interval includes a recorded value on at least 4 of the 7 days of that interval.
Average Daily Posterior Rhinorrhea Score at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
1.8  (0.9) 1.6  (0.9) 1.7  (0.9)
[1]
Measure Description: The Posterior Rhinorrhea Score was assessed daily by the participant via an electronic diary as the response to the following question: Do you feel dripping at the back of the nose? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0=Not at all; 1=Mild; 2=Moderate; and 3=Severe. Baseline was defined as the average of the daily values recorded during the 7-day interval ending on the latest day prior to randomization such that the prior 7-day interval includes a recorded value on at least 4 of the 7 days of that interval.
Average Daily Anterior Rhinorrhea Score at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
1.9  (0.8) 1.9  (0.9) 1.9  (0.9)
[1]
Measure Description: The Anterior Rhinorrhea Score was assessed daily by the participant via an electronic diary as the response to the following question: Do you have a runny nose? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0=Not at all; 1=Mild; 2=Moderate; and 3=Severe. Baseline was defined as the average of the daily values recorded during the 7-day interval ending on the latest day prior to randomization such that the prior 7-day interval includes a recorded value on at least 4 of the 7 days of that interval.
Total Sino-Nasal Outcome Test-22 (SNOT-22) Score at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 62 participants 127 participants
59.8  (18.2) 59.2  (20.5) 59.5  (19.3)
[1]
Measure Description: The SNOT-22 Questionnaire, a disease specific HRQoL measure, comprises a list of 22 symptoms and social or emotional consequences of the nasal disorder. Every participant was asked to rate how severe each problem had been for them over the past 2 weeks on a scale from 0 (no problem at all) to 5 (problem as bad as it can be). The total score is the sum of the scores for all 22 items, ranging from 0 to 110, with a lower score indicating less disease and better HRQoL. Baseline was defined as the last assessment on or before the date of randomization.
University of Pennsylvania Smell Identification Test (UPSIT) Score at Baseline   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 65 participants 61 participants 126 participants
13.1  (7.3) 12.8  (7.6) 13.0  (7.4)
[1]
Measure Description: The UPSIT is a 40-question instrument that measures an individual's ability to detect odors and ranges from 0 to 40, with a higher score indicating a better sense of smell. It is a self-administered "scratch-and-sniff" test provided in booklets that have 40 microencapsulated odorants, each with a multiple-choice option for the response. The number of correct responses is summed to provide a total score. Baseline was defined as the last assessment on or before the date of randomization.
[2]
Measure Analysis Population Description: A baseline UPSIT score was not collected for one participant from the Omalizumab arm because of site error.
Mometasone Prescribed Daily Dose at Baseline  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 62 participants 127 participants
200 micrograms
5
   7.7%
2
   3.2%
7
   5.5%
400 micrograms
60
  92.3%
60
  96.8%
120
  94.5%
1.Primary Outcome
Title Change From Baseline in Nasal Polyp Score (NPS) at Week 24
Hide Description Total NPS ranges from 0 to 8 (sum of 0-4 for left and right nasal passage scores per the following criteria), with a lower score indicating smaller-sized nasal polyps: 0 = No polyps; 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; 2 = Polyps reaching below the lower border of the middle turbinate (modified to accommodate those with a middle turbinectomy, such that polyp must have reached the top of the inferior turbinate.); 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; and 4 = Large polyps causing complete obstruction of the inferior nasal cavity. Two blinded primary independent expert readers reviewed every post-screening recorded video endoscopy for a given participant to determine total NPS. A third reader chose one of the two scores to be used for analysis in cases where there was any discrepancy in total NPS assigned between the two primary readers.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.31
(-0.63 to 0.01)
-0.90
(-1.23 to -0.57)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The primary analysis tested the null hypothesis that no treatment group difference existed for change from baseline in NPS at Week 24. As NPS and NCS are co-primary outcome measures, both null hypotheses for NPS and NCS must be rejected, with parameter estimates indicating a benefit of omalizumab over placebo, for the study to be deemed positive.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0140
Comments Tested at the two-sided 0.05 significance level. There was no adjustment for multiplicity for the co-primary outcome measures.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline NPS, treatment and baseline NPS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.59
Confidence Interval (2-Sided) 95%
-1.05 to -0.12
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.23
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
2.Primary Outcome
Title Change From Baseline in Average Daily Nasal Congestion Score (NCS) at Week 24
Hide Description The Nasal Congestion Score (NCS) was assessed daily by the participant via an electronic diary as the response to the following question: Is your nose blocked? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 24 (Study Days 155 to 186)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.20
(-0.42 to 0.01)
-0.70
(-0.92 to -0.48)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The primary analysis tested the null hypothesis that no treatment group difference existed for change from baseline in NCS at Week 24. As NPS and NCS are co-primary outcome measures, both null hypotheses for NPS and NCS must be rejected, with parameter estimates indicating a benefit of omalizumab over placebo, for the study to be deemed positive.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0017
Comments Tested at the two-sided 0.05 significance level. There was no adjustment for multiplicity for the co-primary outcome measures.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline NCS, treatment and baseline NCS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.50
Confidence Interval (2-Sided) 95%
-0.80 to -0.19
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.15
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
3.Secondary Outcome
Title Change From Baseline in Average Daily Sense of Smell Score at Week 24
Hide Description The Sense of Smell Score was assessed daily by the participant via an electronic diary as the response to the following question: Is your sense of smell reduced? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 24 (Study Days 155 to 186)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.13
(-0.33 to 0.06)
-0.58
(-0.78 to -0.38)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the average daily Sense of Smell Score (SSS) at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0024
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline SSS, treatment and baseline SSS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.45
Confidence Interval (2-Sided) 95%
-0.73 to -0.16
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.14
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
4.Secondary Outcome
Title Change From Baseline in Average Daily Posterior Rhinorrhea Score at Week 24
Hide Description The Posterior Rhinorrhea Score was assessed daily by the participant via an electronic diary as the response to the following question: Do you feel dripping at the back of the nose? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0=Not at all; 1=Mild; 2=Moderate; and 3=Severe. For each study day, a score was calculated using an average of the prior 7 days among available days within a pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days, otherwise the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 24 (Study Days 155 to 186)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.00
(-0.19 to 0.18)
-0.55
(-0.74 to -0.35)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the average daily Posterior Rhinorrhea Score (PRS) at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline PRS, treatment and baseline PRS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.54
Confidence Interval (2-Sided) 95%
-0.81 to -0.27
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.14
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
5.Secondary Outcome
Title Change From Baseline in Nasal Polyp Score (NPS) at Week 16
Hide Description Total NPS ranges from 0 to 8 (sum of 0-4 for left and right nasal passage scores per the following criteria), with a lower score indicating smaller-sized nasal polyps: 0 = No polyps; 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate; 2 = Polyps reaching below the lower border of the middle turbinate (modified to accommodate those with a middle turbinectomy, such that polyp must have reached the top of the inferior turbinate.); 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate; and 4 = Large polyps causing complete obstruction of the inferior nasal cavity. Two blinded primary independent expert readers reviewed every post-screening recorded video endoscopy for a given participant to determine total NPS. A third reader chose one of the two scores to be used for analysis in cases where there was any discrepancy in total NPS assigned between the two primary readers.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 16 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.29
(-0.61 to 0.04)
-1.20
(-1.54 to -0.86)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the NPS at Week 16.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline NPS, treatment and baseline NPS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.91
Confidence Interval (2-Sided) 95%
-1.39 to -0.44
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.24
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
6.Secondary Outcome
Title Change From Baseline in Average Daily Nasal Congestion Score at Week 16
Hide Description The Nasal Congestion Score (NCS) was assessed daily by the participant via an electronic diary as the response to the following question: Is your nose blocked? The four available response options, scored from 0 (no symptoms) to 3 (severe symptoms) were: 0 = Not at all; 1 = Mild; 2 = Moderate; and 3 = Severe. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 16: Study Days 99 to 126), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 112), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 16 (Study Days 99 to 126)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 16 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.21
(-0.41 to -0.01)
-0.80
(-1.00 to -0.59)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the average daily NCS at Week 16.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline NCS, treatment and baseline NCS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.59
Confidence Interval (2-Sided) 95%
-0.87 to -0.30
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.14
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
7.Secondary Outcome
Title Change From Baseline in Participant Reported Health-Related Quality of Life (HRQoL) as Assessed by the Total Sino-Nasal Outcome Test (SNOT)-22 Questionnaire at Week 24
Hide Description The SNOT-22 Questionnaire, a disease specific HRQoL measure, comprises a list of 22 symptoms and social or emotional consequences of the nasal disorder. Every participant was asked to rate how severe each problem had been for them over the past 2 weeks on a scale from 0 (no problem at all) to 5 (problem as bad as it can be). The total score is the sum of the scores for all 22 items, ranging from 0 to 110, with a lower score indicating less disease and better HRQoL. A negative score indicates a decrease (or improvement) from the baseline score.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-6.55
(-10.88 to -2.23)
-21.59
(-26.05 to -17.14)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the SNOT-22 score at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline, treatment and baseline by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -15.04
Confidence Interval (2-Sided) 95%
-21.26 to -8.82
Parameter Dispersion
Type: Standard Error of the Mean
Value: 3.14
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
8.Secondary Outcome
Title Change From Baseline in Average Daily Anterior Rhinorrhea Score at Week 24
Hide Description The Anterior Rhinorrhea Score was assessed daily by the participant via an electronic diary as the response to the following question: Do you have a runny nose? The four available response options were scored from 0 (no symptoms) to 3 (severe symptoms): 0=Not at all; 1=Mild; 2=Moderate; and 3=Severe. For each study day, a score was calculated using an average of the prior 7 days among available days within a pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days, otherwise the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 24 (Study Days 155 to 186)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.08
(-0.27 to 0.11)
-0.70
(-0.90 to -0.51)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the average daily Anterior Rhinorrhea Score (ARS) at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline ARS, treatment and baseline ARS by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -0.63
Confidence Interval (2-Sided) 95%
-0.90 to -0.35
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.14
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
9.Secondary Outcome
Title Number of Participants Requiring Rescue Medication (Systemic Corticosteroids for ≥3 Consecutive Days) Through Week 24
Hide Description A participant was considered to have had the event of requiring rescue medication if they had taken systemic corticosteroids for 3 or more consecutive days at any point between randomization and Week 24; if the participant had greater than 155 days of follow-up on study and had not taken systemic corticosteroids for 3 or more consecutive days, then they did not have the event. Participants with less than 155 days of follow-up on the study were classified as having had the event if they discontinued study drug due to adverse event, progressive disease, or lack of efficacy and remained missing; if the participant had less than 155 days of follow-up on study and had not already met these criteria, they were classified as having a missing outcome. The null hypothesis was to be assessed by the Wald Chi-square test of the treatment term in the logistic regression model. If model convergence was an issue, then Fisher's Exact test was to be used.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants on study through Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Measure Type: Count of Participants
Unit of Measure: Participants
5
   7.7%
1
   1.6%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for requirement of rescue medication through Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1594
Comments Tested at the two-sided 0.05 significance level.
Method Wald Chi-Square
Comments Adjusted for geographic region and asthma/aspirin sensitivity comorbidity status.
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.20
Confidence Interval (2-Sided) 95%
0.02 to 1.89
Estimation Comments Odds ratio is comparing Omalizumab arm to Placebo arm.
10.Secondary Outcome
Title Number of Participants Having Had Surgery for Nasal Polyps Through Week 24
Hide Description A participant was considered to have had the event of surgery for nasal polyps if they underwent the procedure at any point between randomization and Week 24; if the participant had greater than 155 days of follow-up on study and had not undergone surgery for nasal polyps, then they did not have the event. Participants with less than 155 days of follow-up on the study were classified as having had the event if they discontinued study drug due to adverse event, progressive disease, or lack of efficacy and remained missing; if the participant had less than 155 days of follow-up on study and had not already met these criteria, they were classified as having a missing outcome. The null hypothesis was to be assessed by the Wald Chi-square test of the treatment term in the logistic regression model. If model convergence was an issue, then Fisher's Exact test was to be used.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants on study through Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Measure Type: Count of Participants
Unit of Measure: Participants
1
   1.5%
0
   0.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for having had surgery for nasal polyps through Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1.0000
Comments Tested at the two-sided 0.05 significance level.
Method Fisher Exact
Comments Unadjusted
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
0.00 to 20.61
Estimation Comments Odds ratio is comparing Omalizumab arm to Placebo arm.
11.Secondary Outcome
Title Number of Participants With a Change From Baseline at Week 24 in Asthma Quality of Life Questionnaire (AQLQ) of ≥0.5 in Participants With Comorbid Asthma Only
Hide Description The AQLQ is a 32-item participant-reported measure of asthma-related quality of life (QoL) with a total score (the mean of all 32 responses) ranging from 1 (severely impaired) to 7 (not impaired at all); a higher score indicates a better QoL. An increase of at least 0.5 points in the AQLQ score was considered the minimal important difference for improvement in QoL.
Time Frame Baseline and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was conducted only in the subgroup of participants with comorbid asthma at screening and AQLQ assessments at Baseline and Week 24.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 39 38
Measure Type: Count of Participants
Unit of Measure: Participants
12
  30.8%
20
  52.6%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for number of participants with a change from baseline in AQLQ score of ≥0.5 at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0396
Comments [Not Specified]
Method Wald Chi-Square
Comments Adjusted for Baseline AQLQ, geographic region, and aspirin sensitivity status.
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 4.04
Confidence Interval (2-Sided) 95%
1.07 to 15.25
Estimation Comments Odds ratio is comparing Omalizumab arm to Placebo arm.
12.Secondary Outcome
Title Number of Participants Requiring Rescue Treatment (Systemic Corticosteroids For ≥3 Consecutive Days or Having Had Surgery for Nasal Polyps) Through Week 24
Hide Description A participant was considered to have had the event of requiring rescue treatment if they had taken systemic corticosteroids for 3 or more consecutive days or had nasal polypectomy at any point between randomization and Week 24; if the participant had greater than 155 days of follow-up on study and had not received rescue treatment, then they did not have the event. Participants with less than 155 days of follow-up on the study were classified as having had the event if they discontinued study drug due to adverse event, progressive disease, or lack of efficacy and remained missing; if the participant had less than 155 days of follow-up on study and had not already met these criteria, they were classified as having a missing outcome. The null hypothesis was to be assessed by the Wald Chi-square test of the treatment term in the logistic regression model. If model convergence was an issue, then Fisher's Exact test was to be used.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants on study through Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Measure Type: Count of Participants
Unit of Measure: Participants
5
   7.7%
1
   1.6%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for requirement of rescue treatment through Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1594
Comments Tested at the two-sided 0.05 significance level.
Method Wald Chi-Square
Comments Adjusted for geographic region and asthma/aspirin sensitivity comorbidity status.
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.20
Confidence Interval (2-Sided) 95%
0.02 to 1.89
Estimation Comments Odds ratio is comparing Omalizumab arm to Placebo arm.
13.Secondary Outcome
Title Number of Participants With Reduction in the Need for Surgery for Nasal Polyps by Week 24, as Defined by an NPS of ≤4 (Unilateral Score of ≤2 on Each Side) and Improvement in SNOT-22 Score of ≥8.9
Hide Description A participant was considered to have had the event of reduction in the need for surgery for nasal polyps if they had a Nasal Polyp Score (NPS) of ≤4 and an improvement in the SNOT-22 score of ≥8.9 (minimal important difference) without rescue treatment at Week 24; if the participant had received rescue treatment or had discontinued study drug due to adverse event, progressive disease, or lack of efficacy and remained missing, then they did not have the event. Participants without an intercurrent event and without valid Week 24 assessments of both NPS and SNOT-22 were classified as having a missing outcome. The null hypothesis was to be assessed by the Wald Chi-square test of the treatment term in the logistic regression model. If model convergence was an issue, then Fisher's Exact test was to be used.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants on study through Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Measure Type: Count of Participants
Unit of Measure: Participants
2
   3.1%
10
  16.1%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in reduction in the need for surgery for nasal polyps by Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0139
Comments Tested at the two-sided 0.05 significance level.
Method Fisher Exact
Comments Unadjusted
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 6.22
Confidence Interval (2-Sided) 95%
1.23 to 60.23
Estimation Comments Odds ratio is comparing Omalizumab arm to Placebo arm.
14.Secondary Outcome
Title Change From Baseline in Average Daily Total Nasal Symptom Score (TNSS) at Week 24
Hide Description The Total Nasal Symptom Score (TNSS) was defined as the sum of the four individual scores for Nasal Congestion Score, Anterior Rhinorrhea Score, Posterior Rhinorrhea Score, and Sense of Smell Score, ranging from 0 (no symptoms) to 12 (most severe symptoms), assessed daily by the participant via an electronic diary. For each study day, a score was calculated using an average of the prior 7 days among the available days within the pre-specified window (For Week 24: Study Days 155 to 186), excluding the study day itself, if a value had been recorded by the participant on at least 4 of the prior 7 days; otherwise, the 7-day prior average for that study day was to be considered missing. One calculated (non-missing) 7-day prior average was selected for analysis according to the study day with nearest proximity to Week 24 (Study Day 168), with the earlier selected in the case of a tie. Baseline was defined as the (non-missing) 7-day interval ending on the latest day prior to randomization.
Time Frame Baseline, Week 24 (Study Days 155 to 186)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
-0.44
(-1.07 to 0.19)
-2.53
(-3.18 to -1.89)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the average daily Total Nasal Symptom Score (TNSS) at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline TNSS, treatment and baseline by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value -2.09
Confidence Interval (2-Sided) 95%
-3.00 to -1.18
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.46
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm.
15.Secondary Outcome
Title Change From Baseline in Sense of Smell, as Assessed by The University of Pennsylvania Smell Identification Test (UPSIT) Score at Week 24
Hide Description The UPSIT is a 40-question instrument that measures an individual's ability to detect odors and ranges from 0 to 40, with a higher score indicating a better sense of smell. It is a self-administered "scratch-and-sniff" test provided in booklets that have 40 microencapsulated odorants, each with a multiple-choice option for the response. The number of correct responses is summed to provide a total score.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set: all participants randomized to study treatment. Only participants with assessments at Baseline and Week 24 were included in the analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 65 62
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Score on a scale
0.44
(-1.15 to 2.04)
4.31
(2.66 to 5.95)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Omalizumab
Comments The null hypothesis was that no difference exists between the treatment groups for change from baseline in the UPSIT score at Week 24.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0011
Comments Tested at the two-sided 0.05 significance level.
Method Mixed-Effect Model of Repeated Measures
Comments Adjustments: geographic region, asthma/aspirin sensitivity, treatment, timepoint, baseline UPSIT, treatment and baseline by timepoint interactions.
Method of Estimation Estimation Parameter Difference in Least Squares Means
Estimated Value 3.86
Confidence Interval (2-Sided) 95%
1.57 to 6.15
Parameter Dispersion
Type: Standard Error of the Mean
Value: 1.16
Estimation Comments Difference in least squares means: Omalizumab arm minus Placebo arm
16.Secondary Outcome
Title Number of Participants Who Experienced at Least One Adverse Event by Greatest Severity
Hide Description All adverse events (AE) were treatment emergent AEs, defined as any new AE or any worsening of an existing condition with an onset date on or after the first study drug administration date. AEs were assessed for severity according to the following grading scale: mild (discomfort noticed, but no disruption of normal daily activity), moderate (discomfort sufficient to reduce or affect normal daily activity), or severe (incapacitating with inability to work or to perform normal daily activity). The terms "severe" and "serious" are not synonymous; regardless of severity, some events may have also met seriousness criteria. Multiple occurrences of the same AE in one individual are counted once at the greatest intensity.
Time Frame Up to Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: all participants who received at least one dose of study drug, grouped according to treatment received during the treatment period. One participant in the Placebo arm received incorrectly one dose of omalizumab and was included in the Omalizumab arm for safety analyses.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 64 63
Measure Type: Count of Participants
Unit of Measure: Participants
AEs of Any Severity
35
  54.7%
32
  50.8%
Mild AEs
19
  29.7%
18
  28.6%
Moderate AEs
13
  20.3%
10
  15.9%
Severe AEs
3
   4.7%
4
   6.3%
17.Secondary Outcome
Title Number of Participants Who Experienced at Least One Serious Adverse Event
Hide Description A serious adverse event was defined as any adverse event that met any of the following criteria: was fatal; was life-threatening; required or prolonged inpatient hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the study drug; or, was a significant medical event in the investigator's judgment. Multiple occurrences of the same serious adverse event in one individual were counted once.
Time Frame Up to Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: all participants who received at least one dose of study drug, grouped according to treatment received during the treatment period. One participant in the Placebo arm received incorrectly one dose of omalizumab and was included in the Omalizumab arm for safety analyses.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 64 63
Measure Type: Count of Participants
Unit of Measure: Participants
1
   1.6%
3
   4.8%
18.Secondary Outcome
Title Number of Participants With Adverse Events Leading to Omalizumab/Placebo Discontinuation
Hide Description [Not Specified]
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: all participants who received at least one dose of study drug, grouped according to treatment received during the treatment period. One participant in the Placebo arm received incorrectly one dose of omalizumab and was included in the Omalizumab arm for safety analyses.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 64 63
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
19.Secondary Outcome
Title Number of Participants With Laboratory Abnormalities by Highest Grade Post-Baseline
Hide Description Clinical laboratory tests for serum chemistry and hematology parameters were performed at laboratories; any abnormal values (High or Low) were based on laboratory normal ranges. Laboratory abnormalities are presented by the highest grade according to the World Health Organization (WHO) grade for Adverse Events, except for eosinophils and white blood cells that were graded according to the FDA Toxicity Grading Scale for Healthy Volunteers. Not every abnormal laboratory value qualified as an adverse event, only if it met any of the following criteria: clinically significant (per investigator); accompanied by clinical symptoms; resulted in a change in study treatment; or required a change in concomitant therapy. SGPT/ALT = serum glutamic-pyruvic transaminase/alanine aminotransferase; SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate aminotransferase
Time Frame Up to Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: all participants who received at least one dose of study drug, grouped according to treatment received during the treatment period. One participant in the Placebo arm received incorrectly one dose of omalizumab and was included in the Omalizumab arm for safety analyses.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 64 63
Measure Type: Count of Participants
Unit of Measure: Participants
Alkaline Phosphatase - High, Any Grade (Gr.) Number Analyzed 62 participants 60 participants
0
   0.0%
0
   0.0%
SGPT/ALT - High, Any Gr. Number Analyzed 61 participants 60 participants
4
   6.6%
1
   1.7%
SGPT/ALT - High, Gr. 1 Number Analyzed 62 participants 60 participants
4
   6.5%
1
   1.7%
SGOT/AST - High, Any Gr. Number Analyzed 61 participants 60 participants
2
   3.3%
0
   0.0%
SGOT/AST - High, Gr. 1 Number Analyzed 61 participants 60 participants
2
   3.3%
0
   0.0%
Creatinine - High, Any Gr. Number Analyzed 62 participants 60 participants
0
   0.0%
0
   0.0%
Eosinophils, Absolute Count (Abs.) - High, Any Gr. Number Analyzed 63 participants 62 participants
17
  27.0%
8
  12.9%
Eosinophils, Abs. - High, Gr. 1 Number Analyzed 63 participants 62 participants
17
  27.0%
8
  12.9%
Hemoglobin - Low, Any Gr. Number Analyzed 63 participants 62 participants
0
   0.0%
0
   0.0%
Hemoglobin - High, Any Gr. Number Analyzed 63 participants 62 participants
0
   0.0%
0
   0.0%
Neutrophils, Segmented (Abs.) - Low, Any Gr. Number Analyzed 63 participants 62 participants
2
   3.2%
2
   3.2%
Neutrophils, Segmented (Abs.) - Low, Gr. 1 Number Analyzed 63 participants 62 participants
2
   3.2%
2
   3.2%
Platelet - Low, Any Gr. Number Analyzed 63 participants 62 participants
0
   0.0%
0
   0.0%
Potassium - Low, Any Gr. Number Analyzed 62 participants 60 participants
0
   0.0%
0
   0.0%
Potassium - High, Any Gr. Number Analyzed 62 participants 60 participants
1
   1.6%
1
   1.7%
Potassium - High, Gr. 1 Number Analyzed 62 participants 60 participants
1
   1.6%
1
   1.7%
Sodium - Low, Any Gr. Number Analyzed 62 participants 60 participants
0
   0.0%
0
   0.0%
Sodium - High, Any Gr. Number Analyzed 62 participants 60 participants
2
   3.2%
2
   3.3%
Sodium - High, Gr. 1 Number Analyzed 62 participants 60 participants
2
   3.2%
2
   3.3%
Bilirubin - High, Any Gr. Number Analyzed 62 participants 58 participants
2
   3.2%
2
   3.4%
Bilirubin - High, Gr. 1 Number Analyzed 62 participants 58 participants
1
   1.6%
1
   1.7%
Bilirubin - High, Gr. 2 Number Analyzed 62 participants 58 participants
1
   1.6%
1
   1.7%
Total Leukocyte Count - Low, Any Gr. Number Analyzed 63 participants 62 participants
2
   3.2%
0
   0.0%
Total Leukocyte Count - Low, Gr. 1 Number Analyzed 63 participants 62 participants
2
   3.2%
0
   0.0%
Total Leukocyte Count - High, Any Gr. Number Analyzed 63 participants 62 participants
4
   6.3%
0
   0.0%
Total Leukocyte Count - High, Gr. 1 Number Analyzed 63 participants 62 participants
3
   4.8%
0
   0.0%
Total Leukocyte Count - High, Gr. 2 Number Analyzed 63 participants 62 participants
1
   1.6%
0
   0.0%
20.Secondary Outcome
Title Mean Serum Concentration of Omalizumab at Specified Timepoints
Hide Description Serum concentrations of omalizumab were quantified using an enzyme-linked immunoabsorbent assay (ELISA) with a lower limit of quantification (LLOQ) of 28.0 nanograms per millilitre (ng/mL). According to the analysis plan, values below the lower limit of quantification (BLQ) were set to 14 ng/mL (i.e. half of LLOQ value). If one-third or fewer of participants had results that were BLQ, then all summary statistics were to be calculated. However, if more than one-third of participants had results that were BLQ, then the mean and standard deviation were non-reportable and only the median and maximum were to be calculated for that timepoint.
Time Frame Predose on Day 1, Week 16, Week 24, Unscheduled Visit (outside of planned study visits, as clinically indicated), Dosing Termination/Early Termination Visit (up to 28 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics Evaluable Analysis Set: includes participants who received study drug per protocol. Only the omalizumab-treated participants with evaluable samples at each timepoint were included in this analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 0 61
Mean (Standard Deviation)
Unit of Measure: nanograms per millilitre (ng/mL)
Day 1 Number Analyzed 0 participants 60 participants
NA [1]   (NA)
Week 16 Number Analyzed 0 participants 58 participants
33600  (25000)
Week 24 Number Analyzed 0 participants 57 participants
36500  (27200)
Unscheduled Visit Number Analyzed 0 participants 2 participants
44600  (49700)
Dosing Termination/Early Termination Visit Number Analyzed 0 participants 2 participants
34800  (29600)
[1]
Per the analysis plan, the mean and standard deviation at Day 1 (before dosing) were non-reportable because more than one-third of participants (all except for one) had results that were below the lower limit of quantification.
21.Secondary Outcome
Title Median Serum Concentration of Omalizumab at Specified Timepoints
Hide Description Serum concentrations of omalizumab were quantified using an enzyme-linked immunoabsorbent assay (ELISA) with a lower limit of quantification (LLOQ) of 28.0 nanograms per millilitre (ng/mL). According to the analysis plan, values below the lower limit of quantification (BLQ) were set to 14 ng/mL (i.e. half of LLOQ value). If one-third or fewer of participants had results that were BLQ, then all summary statistics were to be calculated. However, if more than one-third of participants had results that were BLQ, then the mean and standard deviation were non-reportable and only the median and maximum were to be calculated for that timepoint.
Time Frame Predose on Day 1, Week 16, Week 24, Unscheduled Visit (outside of planned study visits, as clinically indicated), Dosing Termination/Early Termination Visit (up to 28 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics Evaluable Analysis Set: includes participants who received study drug per protocol. Only the omalizumab-treated participants with evaluable samples at each timepoint were included in this analysis.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 0 61
Median (Full Range)
Unit of Measure: nanograms per millilitre (ng/mL)
Day 1 Number Analyzed 0 participants 60 participants
0.00 [1] 
(NA to 49.5)
Week 16 Number Analyzed 0 participants 58 participants
25000
(6820 to 133000)
Week 24 Number Analyzed 0 participants 57 participants
26300
(8930 to 130000)
Unscheduled Visit Number Analyzed 0 participants 2 participants
44600
(9420 to 79700)
Dosing Termination/Early Termination Visit Number Analyzed 0 participants 2 participants
34800
(13900 to 55700)
[1]
Per the analysis plan, the minimum at Day 1 (before dosing) was non-reportable because more than one-third of participants (all except for one) had results that were below the lower limit of quantification.
22.Secondary Outcome
Title Mean Serum Concentration of Total and Free Immunoglobulin E (IgE) at Specified Timepoints
Hide Description Serum concentrations of total immunoglobulin E (IgE) and free IgE were measured throughout the 24-week blinded treatment period, as target engagement biomarkers of omalizumab, using validated quantitative immunoassays with lower limits of quantification of 2 and 0.83 International Units per millilitre (IU/mL), respectively, and upper limits of quantification (ULQ) of 5000 and 62.5 IU/mL, respectively. The free IgE assay had limited range to measure circulating levels of free IgE in the presence of complexes of omalizumab-IgE. According to the analysis plan for the free IgE assay, results above ULQ were set to 62.5 IU/mL. If results for one-third or fewer of the participants were greater than the ULQ, then all summary statistics were to be reported. However, if the results for more than one-third of participants were greater than the ULQ, then only the median, interquartile range and minimum were calculated, and the mean, standard deviation, and maximum were non-reportable.
Time Frame Predose on Day 1, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics Evaluable Analysis Set: includes participants who received study drug per protocol. The number analyzed includes participants with evaluable samples at each timepoint.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 63 61
Mean (Standard Deviation)
Unit of Measure: IU/mL
Total IgE - Day 1 Number Analyzed 63 participants 60 participants
230  (235) 218  (220)
Total IgE - Week 16 Number Analyzed 60 participants 59 participants
215  (172) 695  (608)
Total IgE - Week 24 Number Analyzed 59 participants 58 participants
253  (319) 641  (559)
Free IgE - Day 1 Number Analyzed 63 participants 61 participants
NA [1]   (NA) NA [1]   (NA)
Free IgE - Week 16 Number Analyzed 60 participants 59 participants
NA [2]   (NA) 11.7  (13.9)
Free IgE - Week 24 Number Analyzed 60 participants 58 participants
NA [2]   (NA) 11.6  (13.3)
[1]
The mean and standard deviation were non-reportable because, per the analysis plan, results for more than one-third of participants (n = 34) were greater than the upper limit of quantification of the free IgE assay, which had limited range.
[2]
The mean and standard deviation were non-reportable because, per the analysis plan, results for more than one-third of participants (n = 30) were greater than the upper limit of quantification of the free IgE assay, which had limited range.
23.Secondary Outcome
Title Median Serum Concentration of Total and Free Immunoglobulin E (IgE) at Specified Timepoints
Hide Description Serum concentrations of total immunoglobulin E (IgE) and free IgE were measured throughout the 24-week blinded treatment period, as target engagement biomarkers of omalizumab, using validated quantitative immunoassays with lower limits of quantification of 2 and 0.83 International Units per millilitre (IU/mL), respectively, and upper limits of quantification (ULQ) of 5000 and 62.5 IU/mL, respectively. The free IgE assay had limited range to measure circulating levels of free IgE in the presence of complexes of omalizumab-IgE. According to the analysis plan for the free IgE assay, results above ULQ were set to 62.5 IU/mL. If results for one-third or fewer of the participants were greater than the ULQ, then all summary statistics were to be reported. However, if the results for more than one-third of participants were greater than the ULQ, then only the median, interquartile range and minimum were calculated, and the mean, standard deviation, and maximum were non-reportable.
Time Frame Predose on Day 1, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics Evaluable Analysis Set: includes participants who received study drug per protocol. The number analyzed includes participants with evaluable samples at each timepoint.
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description:
Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
Overall Number of Participants Analyzed 63 61
Median (Inter-Quartile Range)
Unit of Measure: IU/mL
Total IgE - Day 1 Number Analyzed 63 participants 60 participants
147
(82.0 to 333)
135
(80.0 to 277)
Total IgE - Week 16 Number Analyzed 60 participants 59 participants
141
(83.0 to 320)
437
(292 to 1030)
Total IgE - Week 24 Number Analyzed 59 participants 58 participants
147
(74.0 to 315)
440
(279 to 742)
Free IgE - Day 1 Number Analyzed 63 participants 61 participants
62.5
(29.9 to 62.5)
62.5
(26.2 to 62.5)
Free IgE - Week 16 Number Analyzed 60 participants 59 participants
59.6
(31.3 to 62.5)
7.58
(4.71 to 11.9)
Free IgE - Week 24 Number Analyzed 60 participants 58 participants
59.6
(17.7 to 62.5)
7.65
(5.46 to 10.8)
Time Frame From Baseline until end of safety follow-up (up to 28 weeks)
Adverse Event Reporting Description The safety analysis set consisted of all participants who received at least one dose of study drug, grouped according to treatment received during the treatment period. One participant in the placebo arm received incorrectly one dose of omalizumab and was therefore included in the omalizumab arm in the safety analysis set.
 
Arm/Group Title Placebo Omalizumab
Hide Arm/Group Description Participants received matching placebo as a subcutaneous injection once every 2 weeks or once every 4 weeks. The dose and dosing frequency was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy. Participants received omalizumab as a subcutaneous injection once every 2 weeks (q2w) or once every 4 weeks (q4w). The dose (from 75 mg up to 600 mg) and dosing frequency (q2w or q4w) was determined by serum total IgE level and body weight using the study-drug dosing table. All participants were also treated during the entire study with intranasal corticosteroids (mometasone nasal spray) as background therapy.
All-Cause Mortality
Placebo Omalizumab
Affected / at Risk (%) Affected / at Risk (%)
Total   0/64 (0.00%)      0/63 (0.00%)    
Hide Serious Adverse Events
Placebo Omalizumab
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/64 (1.56%)      3/63 (4.76%)    
Infections and infestations     
Pneumonia  1  1/64 (1.56%)  1 0/63 (0.00%)  0
Injury, poisoning and procedural complications     
Hand fracture  1  0/64 (0.00%)  0 1/63 (1.59%)  1
Snake bite  1  0/64 (0.00%)  0 1/63 (1.59%)  1
Respiratory, thoracic and mediastinal disorders     
Asthma  1  0/64 (0.00%)  0 1/63 (1.59%)  1
1
Term from vocabulary, MedDRA version 21.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Omalizumab
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   20/64 (31.25%)      17/63 (26.98%)    
General disorders     
Injection site reaction  1  2/64 (3.13%)  4 5/63 (7.94%)  8
Infections and infestations     
Nasopharyngitis  1  9/64 (14.06%)  10 5/63 (7.94%)  8
Nervous system disorders     
Headache  1  3/64 (4.69%)  7 7/63 (11.11%)  14
Respiratory, thoracic and mediastinal disorders     
Asthma  1  5/64 (7.81%)  8 1/63 (1.59%)  1
Epistaxis  1  1/64 (1.56%)  1 4/63 (6.35%)  5
Nasal polyps  1  4/64 (6.25%)  5 1/63 (1.59%)  1
1
Term from vocabulary, MedDRA version 21.1
Indicates events were collected by systematic assessment
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.
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800-821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03280537    
Other Study ID Numbers: GA39855
2017-001718-28 ( EudraCT Number )
First Submitted: September 11, 2017
First Posted: September 12, 2017
Results First Submitted: February 14, 2020
Results First Posted: March 23, 2020
Last Update Posted: March 23, 2020