Trial Outcomes & Findings for Inner-City Anti-IgE Therapy for Asthma (NCT NCT00377572)

Last Updated: 2017-03-21

Results Overview

Maximum symptom days was calculated as the largest of the following variables: number of days with wheezing, chest tightness, or cough; number of nights of sleep disturbance; and number of days when activities were affected. This symptom scale ranges from 0 to 14 days per a 2-week look-back period. A higher score reflected a greater number of asthma symptoms. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

419 participants

Primary outcome timeframe

Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Results posted on

2017-03-21

Participant Flow

Participant milestones

Participant milestones
Measure	Omalizumab (Xolair) + Conventional Therapy Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total immunoglobulin E (IgE) level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total immunoglobulin E (IgE) level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Overall Study STARTED	208	211
Overall Study COMPLETED	155	157
Overall Study NOT COMPLETED	53	54

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Inner-City Anti-IgE Therapy for Asthma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Total n=419 Participants Total of all reporting groups
Age, Continuous	10.9 years STANDARD_DEVIATION 3.6 • n=5 Participants	10.8 years STANDARD_DEVIATION 3.4 • n=7 Participants	10.8 years STANDARD_DEVIATION 3.5 • n=5 Participants
Sex: Female, Male Female	86 Participants n=5 Participants	91 Participants n=7 Participants	177 Participants n=5 Participants
Sex: Female, Male Male	122 Participants n=5 Participants	120 Participants n=7 Participants	242 Participants n=5 Participants
Region of Enrollment United States	208 participants n=5 Participants	211 participants n=7 Participants	419 participants n=5 Participants
Duration of Asthma at Baseline	7.5 Years STANDARD_DEVIATION 4.0 • n=5 Participants	7.0 Years STANDARD_DEVIATION 3.8 • n=7 Participants	7.3 Years STANDARD_DEVIATION 3.9 • n=5 Participants
Childhood Asthma Control Test (C-ACT) Score	20.5 C-ACT score STANDARD_DEVIATION 3.8 • n=5 Participants	20.7 C-ACT score STANDARD_DEVIATION 3.9 • n=7 Participants	20.6 C-ACT score STANDARD_DEVIATION 3.8 • n=5 Participants
Asthma Control Test (ACT) Score	20.3 ACT score STANDARD_DEVIATION 3.8 • n=5 Participants	20.3 ACT score STANDARD_DEVIATION 3.1 • n=7 Participants	20.3 ACT score STANDARD_DEVIATION 3.5 • n=5 Participants
Maximum Number of Asthma Symptom Days (Previous 2 Weeks)	3.0 Days STANDARD_DEVIATION 3.5 • n=5 Participants	3.1 Days STANDARD_DEVIATION 3.6 • n=7 Participants	3.0 Days STANDARD_DEVIATION 3.5 • n=5 Participants
Missed Number of School Days (Previous 2 Weeks)	0.23 Days STANDARD_DEVIATION 0.76 • n=5 Participants	0.25 Days STANDARD_DEVIATION 0.63 • n=7 Participants	0.24 Days STANDARD_DEVIATION 0.70 • n=5 Participants
Forced Expiratory Volume in 1 Second (FEV1) % Predicted	93 Percent STANDARD_DEVIATION 19 • n=5 Participants	92 Percent STANDARD_DEVIATION 18 • n=7 Participants	93 Percent STANDARD_DEVIATION 18 • n=5 Participants
FEV1/FVC Ratio	77.3 ratio (x 100) STANDARD_DEVIATION 10.0 • n=5 Participants	77.6 ratio (x 100) STANDARD_DEVIATION 9.4 • n=7 Participants	77.4 ratio (x 100) STANDARD_DEVIATION 9.7 • n=5 Participants

PRIMARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Maximum Number of Asthma Symptom Days	1.48 Days Standard Error 0.10	1.96 Days Standard Error 0.10

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

The number of school days missed was available for 307 of the 419 (73%) study participants, of which 152 were in the Omalizumab (Xolair) + Conventional Therapy arm. Source of data: caretaker/participant self-report. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=152 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=155 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Economic Outcome: Comparison of Number of Missed School Days Due to Asthma	0.16 Days Standard Error 0.03	0.25 Days Standard Error 0.03

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

The number of work days missed by the caretaker due to the study participant's asthma was available for 138 of 419 (33%) study participant caretakers. Source of data: caretaker self-report. Data represent an average of those collected in the time period (weeks 12-60).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=69 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=69 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Economic Outcome: Number of Missed Work Days by Caretaker Due to Asthma	0.13 Days Standard Error 0.135	0.43 Days Standard Error 0.121

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of treatment.

Population: Intent-to-treat

The Childhood Asthma Control Test (C-ACT) is a validated tool to assess overall asthma control (over the last 4 weeks) in patients ages 4 to 11 years. Scores can range from 0 to 27. A score of 19 or less is indicative of asthma that is not well controlled. The minimally important difference in C-ACT scores is not defined. C-ACT scores were available as an outcome measure in 236 of the 419 participants, 118 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=118 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=118 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Child Asthma Control Test (C-ACT) Score	23.0 C-ACT score Standard Error 0.21	22.2 C-ACT score Standard Error 0.21

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

The Asthma Control Test (ACT) is a validated tool to assess overall asthma control (over the last 4 weeks) in patients \>= 12 years of age. It is a questionnaire comprised of 5 questions assessing: asthma symptoms, use of rescue medications, and the impact of asthma on everyday functioning. All questions are scored on a 5-point Likert scale, with a higher score indicating better control. All scores were added together to calculate a total score. Total scores can range from 5 to 25. A score of 19 or less is indicative of asthma that is not well controlled. The minimally important difference for ACT is 3 points. ACT scores as an outcome measure were available in 150 of the 419 participants, 77 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=77 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=73 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Asthma Control Test (ACT) Score	22.5 ACT score Standard Error 0.22	22.3 ACT score Standard Error 0.22

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

FEV1 is air volume exhaled in 1 second during spirometry. For the trial, mild asthma is defined as pre-bronchodilator FEV1 ≥80% predicted, requiring no/low-moderate dose of inhaled glucocorticoids; moderate asthma and severe asthma, respectively, as pre-bronchodilator FEV1 \<80% predicted requiring the same glucocorticoids as mild asthma and FEV1 \<80% predicted requiring high-dose inhaled glucocorticoids (with/without continuous oral glucocorticoids) or uncontrolled despite treatment. FEV1 % of predicted is FEV1 converted to a percentage of normal, based on height, weight, and race. FEV1 percent predicted data as an outcome measure were available in 363 of the 419 participants, 190 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=190 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=173 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Forced Expiratory Volume in 1 Second (FEV1) % Predicted	92.6 Percent Standard Error 0.60	91.7 Percent Standard Error 0.64

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

The FEV1 (forced expiratory volume 1))/ FVC (forced vital capacity) ratio is used to evaluate airways obstructions since pure restrictive ventilatory defects cause an equal reduction in the FEV1 and the FVC. An FEV1/FVC ratio below 80% indicates airflow obstruction. Normal FEV1/FVC: 8 - 19 years of age=85%. FEV1/FVC ratio data as an outcome measure were available in 363 of the 419 participants, 190 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=190 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=173 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
FEV1/FVC Ratio	77.3 Ratio (x100) Standard Error 0.36	77.5 Ratio (x100) Standard Error 0.38

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Exhaled nitric oxide is a biomarker of airway inflammation. Measurement (in parts per billion,ppb) of exhaled nitric oxide (eNO) prior to spirometry, employing a technique modified after Silkoff et al (1997) and following American Thoracic Society guidelines for eNO assessment (American Thoracic Society, 1999). Nitric oxide concentrations were measured using a rapid-response chemiluminescent analyzer (NIOX™ System, Aerocrine, Sweden) which has a response time of \< 700 ms for 10-90% full scale. The Food and Drug Administration has approved this device for clinical application in asthma management. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=152 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=145 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Exhaled Nitric Oxide	22.6 ppb Standard Error 1.46	33.0 ppb Standard Error 1.50

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment

Population: Intent-to-treat

Adherence to the study regimen and other asthma treatments, assessed as percent of expected dose taken, by means of study interviews and study physician corroboration every 3 months. Adherence data as an outcome were available in 384 of the 419 participants, 193 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=193 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=191 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Adherence to Asthma Medication	84.6 percentage of expected dose taken Standard Error 1.78	88.6 percentage of expected dose taken Standard Error 1.80

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment

Population: Intent-to-treat

Treatment steps were established, per the National Asthma Education and Prevention Program Expert Panel Report 3 guidelines. Steps 1-2 apply to mild asthma, 3 to moderate asthma, and 4-6 to severe asthma. At Step 0, the recommendation is for no asthma-control medication or albuterol as needed; at 1, budesonide 180 mcg once a day; at 2, budesonide 180 mcg twice a day; at 3, budesonide 360 mcg twice a day; at 4, fluticasone-salmeterol (Advair, GlaxoSmithKline) 250 mcg fluticasone and 50 mcg salmeterol twice a day; at 5, Advair 250 mcg and 50 mcg twice a day plus montelukast once a day; and at 6, Advair 500 mcg and 50 mcg twice a day plus montelukast once a day. (The doses for montelukast are 5 mg per day for those \<=14 years old and 10 mg per day for those \>=15 years.) Data represent an average of those in the time period, where at \>/= 1 value was available in this period and at baseline for a participant; results are model predicted numbers (e.g.,odds ratios converted to percentages).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Prevalence: Treatment Step Level 1 or 2 (Mild Asthma)	43.6 percent prevalence 4.0	26.7 percent prevalence 3.3

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment

Population: Intent-to-treat

Steps were established, per the National Asthma Education and Prevention Program Expert Panel Report 3 guidelines. Steps 1-2 apply to mild asthma, 3 to moderate asthma, and 4-6 to severe asthma. At Step 0, the recommendation is for no asthma-control medication or albuterol as needed; at 1, budesonide 180 mcg once a day; at 2, budesonide 180 mcg twice a day; at 3, budesonide 360 mcg twice a day; at 4, fluticasone-salmeterol (Advair, GlaxoSmithKline) 250 mcg fluticasone and 50 mcg salmeterol twice a day; at 5, Advair 250 mcg and 50 mcg twice a day plus montelukast once a day; and at 6, Advair 500 mcg and 50 mcg twice a day plus montelukast once a day. (The doses for montelukast are 5 mg per day for those \<=14 years old and 10 mg per day for those \>=15 years.) Data represent an average of those in the time period, where at least one value was available in this period and at baseline for a participant. Results values are model predicted numbers,(e.g, odds ratios converted to percentages).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Prevalence: Treatment Step Level 4 Through 6 (Severe Asthma)	31.2 percent prevalence 3.5	50.8 percent prevalence 4.0

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Prescribed dose (mcg/day) of inhaled glucocorticoids to maintain asthma control. The dose of inhaled glucocorticoids was converted to the budesonide-equivalent dose. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Dose Inhaled Corticosteroids (Glucocorticoids)	663 mcg/day Standard Error 23.3	771 mcg/day Standard Error 23.5

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Percent of participants prescribed long-acting beta 2 agonists to maintain asthma control. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant. Results values are model predicted numbers, (e.g.,odds ratios converted to percentages).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Prevalence: Prescribed Rescue Beta 2 Agonists	55.4 percent prevalence 2.4	65.5 percent prevalence 2.5

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Percent participants with \>=1 hospitalizations. A hospitalization is defined as an asthma-related, overnight hospitalization. . Results values are model predicted numbers,(e.g., odds ratios converted to percentages).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Prevalence: Asthma-Related Medical Care Resource Utilization - Hospitalizations	1.5 percent prevalence 0.9	6.3 percent prevalence 1.8

SECONDARY outcome

Timeframe: Weeks 12-60: 12 months of assessments starting 12 weeks after the initiation of study treatment.

Population: Intent-to-treat

Percent participants with \>=1 exacerbations. An exacerbation was defined as a prednisone burst (a minimum of 20 mg per day of prednisone, or the equivalent, taken for any 3 of 5 consecutive days) or hospitalization. Results values are model predicted numbers, (e.g.,odds ratios converted to percentages).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Percent Prevalence: Asthma Exacerbations	30.3 percent prevalence 3.3	48.8 percent prevalence 3.7

SECONDARY outcome

Timeframe: Week 60

Population: Intent-to-treat

Asthma-Specific Quality of Life (QOL) Measure . The PACQLQ is a validated tool that measures limitations and anxieties faced by primary caregivers of children with asthma. Scores are calculated as the mean score within two domains of questions (re: activity limitation and emotional function) and overall scores represent the mean across all questions. The use of the PACQLQ is valid for use in the caretakers of children ages 7 to 17 years of age. Higher scores indicate better quality of life. Minimum possible score is 1 (maximum impairment); maximum possible score is 7 (no impairment). The range of actual scores were a minimum of 2.4 and a maximum of 7. Method: Caretaker self-report. PACQLQ scores were available for 320 of 419 (76%) of study participant caretakers (159 in the Omalizumab (Xolair) + Conventional Therapy arm).

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=159 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=161 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Asthma Caregiver's Quality of Life Questionnaire (PACQLQ) Overall Score	5.7 units on a scale Standard Deviation 1.2	5.8 units on a scale Standard Deviation 1.1

SECONDARY outcome

Timeframe: Week 60

Population: Intent-to-treat

Asthma-specific quality of life (QOL) validated tool designed for children 7 to 17 years of age. PAQLQ measures functional problems that are most troublesome to children with asthma. PAQLQ has 23 questions in 3 domains (activity limitation=5, emotional function=8, symptoms=10). Patients responded to each question on a 7-point Likert scale. Overall PAQLQ score is mean of 23 questions; each domain score is mean of questions in that domain. Minimum possible score is 1 (maximum impairment); maximum possible score is 7 (no impairment). Actual scores ranged from 2.1 to 7. PAQLQ scores were available for 338 of 419 (81%) of study participants, 170 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm.

Outcome measures

Outcome measures
Measure	Omalizumab (Xolair) + Conventional Therapy n=170 Participants Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=168 Participants Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Paediatric Asthma Quality of Life Questionnaire (PAQLQ) Overall Score	6.1 units on a scale Standard Deviation 1.0	6.2 units on a scale Standard Deviation 0.9

Adverse Events

Omalizumab (Xolair) + Conventional Therapy

Serious events: 24 serious events

Other events: 32 other events

Deaths: 0 deaths

Placebo + Conventional Therapy

Serious events: 34 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 participants at risk Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 participants at risk Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Gastrointestinal disorders Vomiting	0.48% 1/208 • Number of events 1	0.00% 0/211
General disorders Pyrexia	0.48% 1/208 • Number of events 1	0.00% 0/211
Hepatobiliary disorders Cholelithiasis	0.48% 1/208 • Number of events 1	0.00% 0/211
Immune system disorders Anaphylactic reaction	0.48% 1/208 • Number of events 1	1.9% 4/211 • Number of events 4
Immune system disorders Food allergy	0.48% 1/208 • Number of events 1	0.00% 0/211
Immune system disorders Hypersensitivity	0.00% 0/208	0.47% 1/211 • Number of events 1
Infections and infestations Appendicitis	0.48% 1/208 • Number of events 1	0.00% 0/211
Infections and infestations Gastroenteritis viral	0.00% 0/208	0.47% 1/211 • Number of events 1
Infections and infestations Pharyngitis	0.48% 1/208 • Number of events 1	0.00% 0/211
Infections and infestations Pneumonia	0.00% 0/208	0.95% 2/211 • Number of events 2
Injury, poisoning and procedural complications Burns second degree	0.48% 1/208 • Number of events 1	0.00% 0/211
Injury, poisoning and procedural complications Hip fracture	0.48% 1/208 • Number of events 1	0.00% 0/211
Metabolism and nutrition disorders Diabetes mellitus insulin-dependent	0.48% 1/208 • Number of events 1	0.47% 1/211 • Number of events 1
Pregnancy, puerperium and perinatal conditions Abortion spontaneous	0.00% 0/208	0.47% 1/211 • Number of events 1
Pregnancy, puerperium and perinatal conditions Intra-uterine death	0.48% 1/208 • Number of events 1	0.00% 0/211
Psychiatric disorders Affective disorder	0.48% 1/208 • Number of events 1	0.00% 0/211
Psychiatric disorders Aggression	0.48% 1/208 • Number of events 1	0.47% 1/211 • Number of events 1
Psychiatric disorders Anger	0.00% 0/208	0.47% 1/211 • Number of events 1
Psychiatric disorders Attention deficit/hyperactivity disorder	0.48% 1/208 • Number of events 1	0.00% 0/211
Psychiatric disorders Bipolar disorder	0.00% 0/208	0.47% 1/211 • Number of events 1
Reproductive system and breast disorders Genital disorder female	0.00% 0/208	0.47% 1/211 • Number of events 1
Reproductive system and breast disorders Vaginal haemorrhage	0.48% 1/208 • Number of events 1	0.00% 0/211
Respiratory, thoracic and mediastinal disorders Adenoidal hypertrophy	0.00% 0/208	0.47% 1/211 • Number of events 1
Respiratory, thoracic and mediastinal disorders Asthma	6.2% 13/208 • Number of events 17	9.5% 20/211 • Number of events 24
Respiratory, thoracic and mediastinal disorders Cough	0.48% 1/208 • Number of events 1	0.00% 0/211
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/208	0.47% 1/211 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	0.48% 1/208 • Number of events 1	0.00% 0/211
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	0.48% 1/208 • Number of events 1	0.00% 0/211
Respiratory, thoracic and mediastinal disorders Sleep apnoea syndrome	0.00% 0/208	0.47% 1/211 • Number of events 1
Respiratory, thoracic and mediastinal disorders Status asthmaticus	0.00% 0/208	0.95% 2/211 • Number of events 2
Respiratory, thoracic and mediastinal disorders Tonsillar haemorrhage	0.00% 0/208	0.47% 1/211 • Number of events 1
Respiratory, thoracic and mediastinal disorders Tonsillar hypertrophy	0.00% 0/208	0.95% 2/211 • Number of events 2
Respiratory, thoracic and mediastinal disorders Wheezing	0.96% 2/208 • Number of events 2	0.00% 0/211
Skin and subcutaneous tissue disorders Urticaria	0.48% 1/208 • Number of events 1	0.00% 0/211
Vascular disorders Flushing	0.00% 0/208	0.47% 1/211 • Number of events 1

Other adverse events

Other adverse events
Measure	Omalizumab (Xolair) + Conventional Therapy n=208 participants at risk Omalizumab was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.	Placebo + Conventional Therapy n=211 participants at risk Placebo was administered subcutaneously every 2 or 4 weeks over a period of 60 weeks to participants classified as having moderate to severe asthma. Doses (mg) and dosing frequency were determined by serum total IgE level (IU/mL) and body weight (kg). Also, participants continued with their conventional asthma therapy according to the National Asthma Education and Prevention Program (NAEPP-II, 2002) guidelines, under the management of an asthma specialist health care provider.
Infections and infestations Respiratory tract infection	6.7% 14/208 • Number of events 27	8.5% 18/211 • Number of events 34
Infections and infestations Upper respiratory tract infection	9.1% 19/208 • Number of events 25	9.5% 20/211 • Number of events 34

Additional Information

Director, Clinical Research Program

DAIT/NIAID

Phone: 301-594-7669

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place