Clinical trial NCT00079937: Efficacy and Safety of Omalizumab in Children (6 - < 12 Years) With Moderate-severe, Inadequately Controlled Allergic Asthma
Official title: A 1 Year, Randomized, Double-blind, Parallel-group, Placebo-controlled, Multicenter Evaluation of Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omalizumab in Children (6 - < 12 Years) With Moderate-severe, Persistent, Inadequately Controlled Allergic Asthma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Asthma
Keywords: allergic
asthma
atopic
omalizumab
immunoglobulin E
IgE
anti-IgE
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2004-03-22
Enrollment: 628
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A substance called immunoglobulin E (IgE), which is naturally produced by our body, has a key role in generating asthma attacks. In patients with allergies, there is an exaggerated production of IgE in response to specific substances such as pollens. Omalizumab is a new drug that inactivates IgE. This study tested the safety and efficacy of omalizumab against asthma attacks in children with allergic asthma.

Detailed description: This study was designed to provide one year efficacy and safety data for subcutaneous (SC) omalizumab, compared to placebo in children (6 to \< 12 years) with moderate to severe persistent asthma who have inadequate asthma control despite treatment according to National Heart, Lung and Blood Institute (NHLBI) step 3 or 4 (at least medium dose inhaled corticosteroids with or without other controller asthma medications).

Eligibility criteria: Inclusion criteria:

* Parent or legal guardian was informed of the study procedures and medications and gave written informed consent.
* Outpatient males and females aged 6 - \< 12 years on study entry, with body weight between 20 and 150 kg.
* Total serum IgE level ≥ 30 to ≤ 1300 IU.
* Diagnosis of allergic asthma ≥ 1 year duration, according to American Thoracic Society (ATS) criteria, and a screening history consistent with clinical features of moderate or severe persistent asthma according to National Heart Lung and Blood Institute (NHLBI) guidelines.
* Positive prick skin test to at least one perennial allergen, documented within the past 2 years or taken at Screening. A radioallergosorbent test (RAST) could have been performed for patients with a borderline skin prick test result after consultation with Novartis clinical personnel.
* Patients with ≥ 12% increase in forced expiratory volume in 1 second (FEV1) over starting value within 30 minutes of taking up to 4 puffs (4x100 µg) salbutamol (albuterol) or nebulized salbutamol up to 5 mg (or equivalent of alternative B2-agonist) documented within the past year, at screening, during the run-in period, or prior to randomization. Patients were not to take their long acting B2-agonist (LABA) medication within 12 hours of reversibility testing.
* Clinical features of moderate or severe persistent asthma (at least step 3) despite therapy at step 3 or 4 (at least medium dose inhaled corticosteroid (ICS) - fluticasone dry-powder inhaler (DPI) ≥ 200 mg/day or equivalent with or without other controller medications).
* Documented history of experiencing asthma exacerbations and demonstrated inadequate symptom control during the last 4 weeks of run-in despite receiving an equivalent dose of fluticasone DPI ≥ 200 mg/day total daily ex-valve dose.

Exclusion criteria:

* Patients who received systemic corticosteroids for reasons other than asthma, beta-adrenergic antagonists by any route, anticholinergics within 24 hours of Screening, methotrexate, gold salts, cyclosporin or troleandomycin, or had received desensitization therapy with less than 3 months of stable maintenance doses prior to Screening.
* Patients with a history of food or drug related severe anaphylactoid or anaphylactic reaction, a history of allergy to antibiotics, with aspirin or other non-steroidal anti-inflammatory drugs (NSAID)-related asthma (unless the NSAID could be avoided), with active lung disease or acute sinusitis/chest infection, elevated serum IgE levels for other reasons, presence/history of a clinically significant uncontrolled systemic disease, cancer, abnormal, electrocardiogram (ECG) in the previous month, or platelets ≤ 100 x 109/L or clinically significant laboratory abnormalities at Screening.

Primary outcomes:
measure: Rate of Clinically Significant Asthma Exacerbations Per Patient in the 24-week Fixed-dose Steroid Treatment Period
timeFrame: Baseline to end of the fixed-dose steroid treatment period (Week 24)
measure: Percentage of Participants With at Least 1 Adverse Event
timeFrame: Baseline to end of the study (Week 68)
Secondary outcomes:
measure: Change in Mean Nocturnal Asthma Symptom Score From Baseline to the End (Last 4 Weeks) of the 24-week Fixed-dose Steroid Treatment Period
timeFrame: Baseline to the end (last 4 weeks) of the 24-week fixed-dose steroid treatment period
measure: Rate of Clinically Significant Asthma Exacerbations Per Patient in the 52-week Treatment Period
timeFrame: Baseline to end of the treatment period (Week 52)
measure: Change in Mean Daily Number of Puffs of Asthma Rescue Medication From Baseline to the End (Last 4 Weeks) of the 24-week Fixed-dose Steroid Treatment Period
timeFrame: Baseline to the end (last 4 weeks) of the 24-week fixed-dose steroid treatment period
measure: Change in Pediatric Asthma Quality of Life Questionnaire (Standardized) [PAQLQ(S)] Scores From Baseline to the End of the 24-week Fixed-dose Steroid Treatment Period (Week 24)
timeFrame: Baseline to the end of the 24-week fixed-dose steroid treatment period (Week 24)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Alabama Allergy and Asthma Center
state: Alabama
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Little Rock
country: United States
facility: Clinical Research Center
state: Arkansas
city: Huntington Beach
country: United States
facility: Allergy and Asthma Specialists Medical Group
state: California
city: Huntington Beach
country: United States
facility: Pediatric Care and Medical Group
state: California
city: Long Beach
country: United States
facility: West Coast Clinical Trials
state: California
city: Mission Viejo
country: United States
facility: Southern California Research Center
state: California
city: Orange
country: United States
facility: Children's Hosptial of Orange County, Div Asthma, Allergy & Immunology
state: California
city: Palmdale
country: United States
facility: CA Allergy & Asthma Med Group
state: California
city: Palo Alto
country: United States
facility: Dr. Joann Blessing-Moore
state: California
city: Riverside
country: United States
facility: Integrated Research Group
state: California
city: San Diego
country: United States
facility: Allergy Associates Medical Group
state: California
city: San Diego
country: United States
facility: Allergy and Asthma Medical Group & Research Center
state: California
city: San Jose
country: United States
facility: Allergy and Asthma Associates of Santa Clara Valley RC
state: California
city: Santa Monica
country: United States
facility: 1304 15th St
state: California
city: Stockton
country: United States
facility: Bensch Research Associates
state: California
city: Walnut Creek
country: United States
facility: Allergy & Asthma Med Group of Diablo Valley CR
state: California
city: Denver
country: United States
facility: National Jewish Medical and Research Center
state: Colorado
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Albany
country: United States
facility: Georgia Pollens
state: Georgia
city: Atlanta
country: United States
facility: Family Allergy and Asthma Center, PC
state: Georgia
city: Savannah
country: United States
facility: Aeroallergy Research Labs of Savannah, Inc
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Elliott City
country: United States
facility: Asthma & Allergy Center
state: Maryland
city: North Dartmouth
country: United States
facility: Northeast Med Research Associates
state: Massachusetts
city: St. Louis
country: United States
facility: St. Louis University School of Medicine
state: Missouri
city: Omaha
country: United States
facility: Midwest Allergy & Asthma Clinic
state: Nebraska
city: Brick
country: United States
facility: Ocean Allergy & Respiratory Research Center
state: New Jersey
city: Newark
country: United States
facility: UMDNJ
state: New Jersey
city: Buffalo
country: United States
facility: Womes And childrens Hospital of Buffalo
state: New York
city: Ithaca
country: United States
facility: Asthma & Allergy Associates
state: New York
city: Liverpool
country: United States
facility: Allergy and Asthma Diagnostic Office
state: New York
city: Rockville Center
country: United States
facility: Island Medical Research (Allergy and Asthma Center)
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: High Point
country: United States
facility: Allergy & Asthma Center of North carolina
state: North Carolina
city: Cincinnati
country: United States
facility: Bernstein Clinical Research Center
state: Ohio
city: Oklahoma City
country: United States
facility: Resp Dis of Children and Adolescents
state: Oklahoma
city: Medford
country: United States
facility: Clinical Research Institute of Southern Oregon
state: Oregon
city: Altoona
country: United States
facility: 501 Howard Av
state: Pennsylvania
city: Pittsburgh
country: United States
facility: West Penn Allegheny General Health System
state: Pennsylvania
city: Upland
country: United States
facility: Asthma and Allergy Associates
state: Pennsylvania
city: Lincoln
country: United States
facility: AAPRI Clinical Research Institute
state: Rhode Island
city: Knoxville
country: United States
facility: Allergy Assoc., The ASthma, Allergy & Sinus Ctr
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: Pediatric Allergy/Immunology Associates, PA
state: Texas
city: Dallas
country: United States
facility: Pediatric Pulmonary Association of North Texas
state: Texas
city: Ft. Worth
country: United States
facility: North Texas Institute for Clinical Trials
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: 7707 Fannin/Ste. 195
state: Texas
city: San Antonio
country: United States
facility: Sylvanna Research
state: Texas
city: South Jordan
country: United States
facility: Copperview Medical Center
state: Utah
city: Norfolk
country: United States
facility: Childrens Hospital of the Kings Daughters
state: Virginia
city: Richmond
country: United States
facility: Virgina Commonwealth
state: Virginia
city: Seattle
country: United States
facility: A.S.T.H.M.A., Inc.
state: Washington
city: Spokane
country: United States
facility: 508 W 6th Av
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Busse WW, Szefler SJ, Haselkorn T, Iqbal A, Ortiz B, Lanier BQ, Chipps BE. Possible Protective Effect of Omalizumab on Lung Function Decline in Patients Experiencing Asthma Exacerbations. J Allergy Clin Immunol Pract. 2021 Mar;9(3):1201-1211. doi: 10.1016/j.jaip.2020.10.027. Epub 2020 Oct 24.
citation: Szefler SJ, Casale TB, Haselkorn T, Yoo B, Ortiz B, Kattan M, Busse WW. Treatment Benefit with Omalizumab in Children by Indicators of Asthma Severity. J Allergy Clin Immunol Pract. 2020 Sep;8(8):2673-2680.e3. doi: 10.1016/j.jaip.2020.03.033. Epub 2020 Apr 13.
citation: Lanier B, Bridges T, Kulus M, Taylor AF, Berhane I, Vidaurre CF. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol. 2009 Dec;124(6):1210-6. doi: 10.1016/j.jaci.2009.09.021.