Trial Outcomes & Findings for Efficacy of Omalizumab in Adults (18-60 Years of Age) With Moderate-Severe, Persistent Allergic Asthma, Despite Receiving Inhaled Corticosteroids and Long Acting Beta-agonists (NCT NCT00670930)
NCT ID: NCT00670930
Last Updated: 2013-01-18
Results Overview
The primary variable of change from baseline in total epithelia eosinophils at end of Week 78 was analyzed on sub-population such as responders and non-responders. Responders are defined as all patients having a Global Evaluation of Treatment Effectiveness (GETE) outcome of excellent or good where as non-responders are with GETE outcome of poor, moderate or worsening. GETE categories are excellent, good, moderate, poor, worsening, and missing as determined by the investigator.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
36 participants
Primary outcome timeframe
Baseline, at end of week 78
Results posted on
2013-01-18
Participant Flow
Participant milestones
| Measure |
Omalizumab
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Overall Study
STARTED
|
23
|
13
|
|
Overall Study
Intent to Treat (ITT) Population
|
23
|
12
|
|
Overall Study
COMPLETED
|
22
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Omalizumab
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Overall Study
Adminstrative Problem
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
Baseline Characteristics
Efficacy of Omalizumab in Adults (18-60 Years of Age) With Moderate-Severe, Persistent Allergic Asthma, Despite Receiving Inhaled Corticosteroids and Long Acting Beta-agonists
Baseline characteristics by cohort
| Measure |
Omalizumab
n=23 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=12 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
43.7 Years
STANDARD_DEVIATION 9.66 • n=5 Participants
|
41.8 Years
STANDARD_DEVIATION 10.43 • n=7 Participants
|
43.1 Years
STANDARD_DEVIATION 9.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, at end of week 78Population: Intent-to-treat (ITT) population: The ITT population consisted of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment they were assigned to at randomization. Patients with both baseline and af end of week 78 data in each category have been reported.
The primary variable of change from baseline in total epithelia eosinophils at end of Week 78 was analyzed on sub-population such as responders and non-responders. Responders are defined as all patients having a Global Evaluation of Treatment Effectiveness (GETE) outcome of excellent or good where as non-responders are with GETE outcome of poor, moderate or worsening. GETE categories are excellent, good, moderate, poor, worsening, and missing as determined by the investigator.
Outcome measures
| Measure |
Omalizumab
n=17 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=11 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Change From Baseline in Total Subepithelial Eosinophils at the End of Week 78 (End of Treatment)
Non-responder (n = 8, 8)
|
1.555 cells/mm^2
Standard Deviation 11.065
|
-5.626 cells/mm^2
Standard Deviation 15.816
|
|
Change From Baseline in Total Subepithelial Eosinophils at the End of Week 78 (End of Treatment)
Responder (n= 9, 3)
|
-5.807 cells/mm^2
Standard Deviation 13.921
|
-5.890 cells/mm^2
Standard Deviation 9.128
|
SECONDARY outcome
Timeframe: Baseline, at end of week 78Population: Intent-to-treat (ITT) population: The ITT population consisted of all randomized patients. Patients with both baseline and end of treatment (at the end of week 78) data were only included for the analysis under each category.
The variable of change from baseline in Sub-epithelial cell count of mast cells at end of Week 78 was analyzed on sub-population such as responders and non-responders. Responders are defined as all patients having a Global Evaluation of Treatment Effectiveness (GETE) outcome of excellent or good where as non-responders are with GETE outcome of poor, moderate or worsening. GETE categories are excellent, good, moderate, poor, worsening, and missing as determined by the investigator.
Outcome measures
| Measure |
Omalizumab
n=17 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=11 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Change From Baseline in Sub-epithelial Cell Count of Mast Cells Following 78 Weeks Treatment, as Assessed Biopsy Samples
Responder (n= 9, 3)
|
-1.392 cells/mm^2
Standard Deviation 13.123
|
5.840 cells/mm^2
Standard Deviation 19.809
|
|
Change From Baseline in Sub-epithelial Cell Count of Mast Cells Following 78 Weeks Treatment, as Assessed Biopsy Samples
Non-responder (n= 8, 8)
|
10.140 cells/mm^2
Standard Deviation 10.266
|
1.114 cells/mm^2
Standard Deviation 18.397
|
SECONDARY outcome
Timeframe: Baseline, at end of week 78Population: Intent-to-treat (ITT) population: The ITT population consisted of all randomized patients. Patients with both baseline and end of treatment (at the end of week 78) data were only included for the analysis under each category.
The variable of change from baseline in Sub-epithelial CD4+ T-lymphocytes at end of Week 78 was analyzed on sub-population such as responders and non-responders. Responders are defined as all patients having a Global Evaluation of Treatment Effectiveness (GETE) outcome of excellent or good where as non-responders are with GETE outcome of poor, moderate or worsening. GETE categories are excellent, good, moderate, poor, worsening, and missing as determined by the investigator.
Outcome measures
| Measure |
Omalizumab
n=17 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=11 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Change From Baseline in Sub-epithelial CD4+ T-lymphocytes Following 78 Weeks Treatment, as Assessed Biopsy Samples
Responder (n= 9, 3)
|
-5.820 cells/mm^2
Standard Deviation 12.490
|
-2.693 cells/mm^2
Standard Deviation 8.117
|
|
Change From Baseline in Sub-epithelial CD4+ T-lymphocytes Following 78 Weeks Treatment, as Assessed Biopsy Samples
Non-responder (n= 8, 8)
|
-4.719 cells/mm^2
Standard Deviation 11.021
|
-7.320 cells/mm^2
Standard Deviation 13.950
|
SECONDARY outcome
Timeframe: Baseline, at end of week 78Population: Intent-to-treat (ITT) population: The ITT population consisted of all randomized patients. Patients with both baseline and end of treatment (at the end of week 78) data were only included for the analysis under each category.
The variable of change from baseline in thickness of the lamina reticularis at end of Week 78 was analyzed on sub-population such as responders and non-responders. Responders are defined as all patients having a Global Evaluation of Treatment Effectiveness (GETE) outcome of excellent or good where as non-responders are with GETE outcome of poor, moderate or worsening. GETE categories are excellent, good, moderate, poor, worsening, and missing as determined by the investigator.
Outcome measures
| Measure |
Omalizumab
n=16 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=11 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Change From Baseline in Thickness of the Lamina Reticularis Following 78 Weeks Treatment, as Assessed Biopsy Samples
Responder (n= 8, 3)
|
-1.300 micrometer(µm)
Standard Deviation 2.926
|
-1.603 micrometer(µm)
Standard Deviation 0.258
|
|
Change From Baseline in Thickness of the Lamina Reticularis Following 78 Weeks Treatment, as Assessed Biopsy Samples
Non-responder (n= 8, 8)
|
0.098 micrometer(µm)
Standard Deviation 2.276
|
-0.659 micrometer(µm)
Standard Deviation 2.288
|
SECONDARY outcome
Timeframe: 78 weeksPopulation: The safety population consisted of all patients in the intent to treat (ITT) population that received at least one dose of study drug and had at least one post-baseline safety assessment.
Outcome measures
| Measure |
Omalizumab
n=23 Participants
Omalizumab was supplied as lyophilized, sterile powder in a single use, 5 ml vial that was designed to deliver 150 mg of omalizumab for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The dose administered was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and the number of injections and injection volume was determined using protocol-specified dosing tables. Omalizumab 75 to 375 mg was administered SQ every 2 or 4 weeks depending on the dose for the 78 weeks duration of double-blinded treatment.
|
Placebo
n=13 Participants
Omalizumab matching placebo was supplied as lyophilized, sterile powder in a single-use, 5 ml vial that was designed to deliver omalizumab matching placebo for subcutaneous (SQ) administration upon reconstitution with 1.4 ml sterile water for injection. The number of injections and injection volume was individualized for each patient based on the patient's body weight and total serum Immunoglobulin E (IgE) level at Visit 1 and was determined using protocol-specified dosing tables. Placebo was administered SQ every 2 or 4 weeks for the 78 weeks duration of double-blinded treatment.
|
|---|---|---|
|
Number of Participants With Adverse Events, Serious Adverse Events and Death as an Assessment of Safety and Tolerability of 78 Weeks Therapy
At least one adverse event
|
19 Participants
|
12 Participants
|
|
Number of Participants With Adverse Events, Serious Adverse Events and Death as an Assessment of Safety and Tolerability of 78 Weeks Therapy
At least one serious adverse event
|
1 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events, Serious Adverse Events and Death as an Assessment of Safety and Tolerability of 78 Weeks Therapy
Death
|
0 Participants
|
0 Participants
|
Adverse Events
Omalizumab
Serious events: 1 serious events
Other events: 16 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Omalizumab
n=23 participants at risk
Omalizumab
|
Placebo
n=13 participants at risk
Placebo
|
|---|---|---|
|
Hepatobiliary disorders
Biliary colic
|
4.3%
1/23
|
0.00%
0/13
|
Other adverse events
| Measure |
Omalizumab
n=23 participants at risk
Omalizumab
|
Placebo
n=13 participants at risk
Placebo
|
|---|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
0.00%
0/23
|
7.7%
1/13
|
|
Cardiac disorders
Palpitations
|
8.7%
2/23
|
0.00%
0/13
|
|
Ear and labyrinth disorders
Tinnitus
|
0.00%
0/23
|
7.7%
1/13
|
|
Eye disorders
Photopsia
|
0.00%
0/23
|
7.7%
1/13
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/23
|
7.7%
1/13
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/23
|
7.7%
1/13
|
|
Gastrointestinal disorders
Diarrhoea
|
4.3%
1/23
|
23.1%
3/13
|
|
Gastrointestinal disorders
Dyspepsia
|
8.7%
2/23
|
0.00%
0/13
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/23
|
7.7%
1/13
|
|
Gastrointestinal disorders
Nausea
|
13.0%
3/23
|
15.4%
2/13
|
|
Gastrointestinal disorders
Vomiting
|
4.3%
1/23
|
7.7%
1/13
|
|
General disorders
Chest pain
|
8.7%
2/23
|
0.00%
0/13
|
|
General disorders
Fatigue
|
8.7%
2/23
|
0.00%
0/13
|
|
General disorders
Injection site erythema
|
8.7%
2/23
|
0.00%
0/13
|
|
General disorders
Injection site rash
|
0.00%
0/23
|
7.7%
1/13
|
|
General disorders
Injection site swelling
|
8.7%
2/23
|
0.00%
0/13
|
|
General disorders
Pain
|
8.7%
2/23
|
0.00%
0/13
|
|
Immune system disorders
Allergy to arthropod bite
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Acute sinusitis
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Alveolar osteitis
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Bronchitis
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Influenza
|
8.7%
2/23
|
15.4%
2/13
|
|
Infections and infestations
Lower respiratory tract infection
|
13.0%
3/23
|
15.4%
2/13
|
|
Infections and infestations
Nasopharyngitis
|
17.4%
4/23
|
15.4%
2/13
|
|
Infections and infestations
Pneumonia
|
13.0%
3/23
|
0.00%
0/13
|
|
Infections and infestations
Sinusitis
|
8.7%
2/23
|
23.1%
3/13
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Tracheobronchitis
|
0.00%
0/23
|
7.7%
1/13
|
|
Infections and infestations
Upper respiratory tract infection
|
13.0%
3/23
|
15.4%
2/13
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
0.00%
0/23
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Concussion
|
0.00%
0/23
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Eye injury
|
0.00%
0/23
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Joint sprain
|
4.3%
1/23
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Muscle injury
|
4.3%
1/23
|
7.7%
1/13
|
|
Injury, poisoning and procedural complications
Muscle rupture
|
0.00%
0/23
|
7.7%
1/13
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/23
|
7.7%
1/13
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/23
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/23
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
13.0%
3/23
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.7%
2/23
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/23
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/23
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
0.00%
0/23
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.7%
2/23
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
0.00%
0/23
|
7.7%
1/13
|
|
Nervous system disorders
Dizziness
|
8.7%
2/23
|
7.7%
1/13
|
|
Nervous system disorders
Headache
|
21.7%
5/23
|
0.00%
0/13
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.7%
2/23
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Increased bronchial secretion
|
0.00%
0/23
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/23
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
4.3%
1/23
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
8.7%
2/23
|
0.00%
0/13
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/23
|
7.7%
1/13
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
- Publication restrictions are in place
Restriction type: OTHER