Trial Outcomes & Findings for A Study Evaluating the Persistency of Response With or Without Xolair (Omalizumab) After Long-term Therapy (NCT NCT01125748)

Last Updated: 2014-10-15

Results Overview

A protocol-defined severe exacerbation was a clinically significant worsening of asthma which, in the clinical judgment of the investigator, required at least 1 of the following: (1) Initiation of systemic corticosteroid treatment (tablets, suspension, or injection) or an increase in the level of systemic corticosteroid treatment from a stable maintenance dose for at least 3 days (For patients taking chronic oral corticosteroids, a protocol-defined severe exacerbation was any clinically significant worsening of asthma requiring ≥ 3 days of treatment with at least a 20 mg increase in the average daily dose of oral prednisone or a comparable dose of systemic corticosteroids) or (2) a hospitalization or emergency room visit because of asthma requiring systemic corticosteroids.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

176 participants

Primary outcome timeframe

Baseline to the end of the study (up to 52 weeks)

Results posted on

2014-10-15

Participant Flow

Participant milestones

Participant milestones
Measure	Omalizumab Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Overall Study STARTED	88	88
Overall Study COMPLETED	78	74
Overall Study NOT COMPLETED	10	14

Reasons for withdrawal

Reasons for withdrawal
Measure	Omalizumab Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Overall Study Adverse Event	0	2
Overall Study Lost to Follow-up	0	1
Overall Study Physician Decision	8	7
Overall Study Patient Decision to Withdraw	2	4

Baseline Characteristics

A Study Evaluating the Persistency of Response With or Without Xolair (Omalizumab) After Long-term Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Omalizumab n=88 Participants Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo n=88 Participants Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.	Total n=176 Participants Total of all reporting groups
Age, Continuous	51.14 years STANDARD_DEVIATION 11.73 • n=5 Participants	51.86 years STANDARD_DEVIATION 13.25 • n=7 Participants	51.50 years STANDARD_DEVIATION 12.48 • n=5 Participants
Sex: Female, Male Female	63 Participants n=5 Participants	60 Participants n=7 Participants	123 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants	28 Participants n=7 Participants	53 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to the end of the study (up to 52 weeks)

Population: Intent-to-treat population: All randomized participants.

Outcome measures

Outcome measures
Measure	Omalizumab n=88 Participants Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo n=88 Participants Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Percentage of Participants Not Experiencing a Protocol-defined Severe Exacerbation During the Study	67.0 Percentage of participants Interval 57.2 to 76.9	47.7 Percentage of participants Interval 37.3 to 58.2

SECONDARY outcome

Timeframe: Baseline to the end of the study (up to 52 weeks)

Population: Intent-to-treat population: All randomized participants.

Outcome measures

Outcome measures
Measure	Omalizumab n=88 Participants Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo n=88 Participants Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Time to the First Protocol-defined Severe Exacerbation	21.7 Weeks Interval 14.41 to 29.03	16.6 Weeks Interval 11.9 to 21.26

Adverse Events

Omalizumab

Serious events: 10 serious events

Other events: 100 other events

Deaths: 0 deaths

Placebo

Serious events: 8 serious events

Other events: 88 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Omalizumab n=121 participants at risk Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo n=88 participants at risk Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Infections and infestations Bronchitis	7.4% 9/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	9.1% 8/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Respiratory, thoracic and mediastinal disorders Acute sinusitis	10.7% 13/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	10.2% 9/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Respiratory, thoracic and mediastinal disorders Nasopharyngitis	2.5% 3/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	5.7% 5/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Respiratory, thoracic and mediastinal disorders Sinusitis	21.5% 26/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	13.6% 12/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract infection	19.0% 23/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	12.5% 11/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Renal and urinary disorders Urinary tract infection	5.0% 6/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	6.8% 6/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Immune system disorders Asthma	23.1% 28/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	34.1% 30/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Immune system disorders Rhinitis allergic	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	8.0% 7/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER

Measure	Omalizumab n=121 participants at risk Participants received omalizumab subcutaneously at the same dose and dosing interval as administered prior to enrollment in this study. The dose of omalizumab was either a minimum of 0.008 mg/kg/IgE (IU/mL) every 2 weeks or a minimum of 0.016 mg/kg/IgE (IU/mL) every 4 weeks for 48 weeks.	Placebo n=88 participants at risk Participants received placebo subcutaneously at the same dosing interval as omalizumab was administered prior to enrollment in this study.
Blood and lymphatic system disorders Spontaneous haematoma	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Gastrointestinal disorders Dyskinesia oesophageal	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Gastrointestinal disorders Dysphagia	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Gastrointestinal disorders Eosinophilic oesophagitis	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Hepatobiliary disorders Cholecystitis	0.00% 0/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	1.1% 1/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Infections and infestations Upper respiratory tract infection	0.00% 0/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	1.1% 1/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Injury, poisoning and procedural complications Laceration	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Musculoskeletal and connective tissue disorders Intervertebral disc degeneration	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Musculoskeletal and connective tissue disorders Osteoarthritis	0.00% 0/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	1.1% 1/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adenocarcinoma of colon	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Mueller's mixed tumour	0.00% 0/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	1.1% 1/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Nervous system disorders Syncope	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Psychiatric disorders Anxiety	0.00% 0/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	1.1% 1/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Respiratory, thoracic and mediastinal disorders Asthma	5.0% 6/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	4.5% 4/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.
Vascular disorders Haematoma	0.83% 1/121 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.	0.00% 0/88 Safety population: All randomized participants who had at least 1 injection. Adverse events for participants who crossed-over from placebo to Xolair are included in the Xolair Continuation group. Participants randomized to placebo who switched before 52 weeks are included in both columns; their adverse events are only reported once.