Efficacy and Safety of Omalizumab in Children (6 - < 12 Years) With Moderate-severe, Inadequately Controlled Allergic Asthma

NCT ID: NCT00079937

Last Updated: 2012-04-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 628 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-04-30
• Study Completion Date: 2008-03-31

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).

Conditions

Asthma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Omalizumab

Participants received omalizumab administered by subcutaneous injection every 2 or 4 weeks for a duration of 52 weeks. The omalizumab dose was based on the patient's body weight and total serum IgE level at Screening. The first 24 weeks of the treatment period was a fixed steroid phase where the steroid dose was maintained constant; in the following 28 weeks the steroid dose was adjustable, depending on the patient's condition. Following the 52-week treatment period, patients were followed up for an additional 16 weeks.

Group Type: EXPERIMENTAL

Omalizumab

Intervention Type: DRUG

The omalizumab dose administered, based on the patient's body weight and total serum IgE level at Screening, and the number of injections and injection volume was determined from the dosing tables in the protocol. Omalizumab 75 to 375 mg was administered subcutaneous (SC) every 2 or 4 weeks depending on the dose.

Fluticasone

Intervention Type: DRUG

Patients entered the study using their current formulation of any inhaled steroid (proprietary drug and device) ≥ 200 μg/day equivalent of fluticasone administered with a dry-powder inhaler.

Placebo

Placebo was administered by subcutaneous injection every 2 or 4 weeks depending on the dosing schedule in the protocol for a total of 52 weeks. The first 24 weeks of the treatment period was a fixed steroid phase where the steroid dose was maintained constant; in the following 28 weeks the steroid dose was adjustable, depending on the patient's condition. Following the 52-week treatment period, patients were followed up for an additional 16 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo was administered subcutaneous (SC) every 2 or 4 weeks depending on the dosing schedule in the protocol.

Fluticasone

Intervention Type: DRUG

Patients entered the study using their current formulation of any inhaled steroid (proprietary drug and device) ≥ 200 μg/day equivalent of fluticasone administered with a dry-powder inhaler.

Interventions

Omalizumab

The omalizumab dose administered, based on the patient's body weight and total serum IgE level at Screening, and the number of injections and injection volume was determined from the dosing tables in the protocol. Omalizumab 75 to 375 mg was administered subcutaneous (SC) every 2 or 4 weeks depending on the dose.

Intervention Type: DRUG

Placebo

Placebo was administered subcutaneous (SC) every 2 or 4 weeks depending on the dosing schedule in the protocol.

Intervention Type: DRUG

Fluticasone

Patients entered the study using their current formulation of any inhaled steroid (proprietary drug and device) ≥ 200 μg/day equivalent of fluticasone administered with a dry-powder inhaler.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Alabama Allergy and Asthma Center

Birmingham, Alabama, United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Clinical Research Center

Little Rock, Arkansas, United States

Allergy and Asthma Specialists Medical Group

Huntington Beach, California, United States

Pediatric Care and Medical Group

Huntington Beach, California, United States

West Coast Clinical Trials

Long Beach, California, United States

Southern California Research Center

Mission Viejo, California, United States

Children's Hosptial of Orange County, Div Asthma, Allergy & Immunology

Orange, California, United States

CA Allergy & Asthma Med Group

Palmdale, California, United States

Dr. Joann Blessing-Moore

Palo Alto, California, United States

Integrated Research Group

Riverside, California, United States

Allergy Associates Medical Group

San Diego, California, United States

Allergy and Asthma Medical Group & Research Center

San Diego, California, United States

Allergy and Asthma Associates of Santa Clara Valley RC

San Jose, California, United States

1304 15th St

Santa Monica, California, United States

Bensch Research Associates

Stockton, California, United States

Allergy & Asthma Med Group of Diablo Valley CR

Walnut Creek, California, United States

National Jewish Medical and Research Center

Denver, Colorado, United States

Miami Children's Hospital

Miami, Florida, United States

Georgia Pollens

Albany, Georgia, United States

Family Allergy and Asthma Center, PC

Atlanta, Georgia, United States

Aeroallergy Research Labs of Savannah, Inc

Savannah, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Asthma & Allergy Center

Elliott, Maryland, United States

Northeast Med Research Associates

North Dartmouth, Massachusetts, United States

St. Louis University School of Medicine

St Louis, Missouri, United States

Midwest Allergy & Asthma Clinic

Omaha, Nebraska, United States

Ocean Allergy & Respiratory Research Center

Brick, New Jersey, United States

UMDNJ

Newark, New Jersey, United States

Womes And childrens Hospital of Buffalo

Buffalo, New York, United States

Asthma & Allergy Associates

Ithaca, New York, United States

Allergy and Asthma Diagnostic Office

Liverpool, New York, United States

Island Medical Research (Allergy and Asthma Center)

Rockville Centre, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Allergy & Asthma Center of North carolina

High Point, North Carolina, United States

Bernstein Clinical Research Center

Cincinnati, Ohio, United States

Resp Dis of Children and Adolescents

Oklahoma City, Oklahoma, United States

Clinical Research Institute of Southern Oregon

Medford, Oregon, United States

501 Howard Av

Altoona, Pennsylvania, United States

West Penn Allegheny General Health System

Pittsburgh, Pennsylvania, United States

Asthma and Allergy Associates

Upland, Pennsylvania, United States

AAPRI Clinical Research Institute

Lincoln, Rhode Island, United States

Allergy Assoc., The ASthma, Allergy & Sinus Ctr

Knoxville, Tennessee, United States

Vanderbilt University

Nashville, Tennessee, United States

Pediatric Allergy/Immunology Associates, PA

Dallas, Texas, United States

Pediatric Pulmonary Association of North Texas

Dallas, Texas, United States

North Texas Institute for Clinical Trials

Fort Worth, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

7707 Fannin/Ste. 195

Houston, Texas, United States

Sylvanna Research

San Antonio, Texas, United States

Copperview Medical Center

South Jordan, Utah, United States

Childrens Hospital of the Kings Daughters

Norfolk, Virginia, United States

Virgina Commonwealth

Richmond, Virginia, United States

A.S.T.H.M.A., Inc.

Seattle, Washington, United States

508 W 6th Av

Spokane, Washington, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

References

Fiocchi A, Chinthrajah RS, Ansotegui IJ, Sriaroon P, Mustafa SS, Raut P, Cameron B, Gupta S, Fleischer DM. Does Comorbid Food Allergy Affect Response to Omalizumab in Patients with Asthma? J Asthma Allergy. 2024 Sep 17;17:889-900. doi: 10.2147/JAA.S475517. eCollection 2024.

Reference Type: DERIVED

PMID: 39309477 (View on PubMed)

Witonsky J, Elhawary JR, Millette LA, Holweg CTJ, Ko J, Raut P, Borrell LN. Similar response to omalizumab in children with allergic asthma from different racial backgrounds. J Allergy Clin Immunol Pract. 2023 Sep;11(9):2911-2913. doi: 10.1016/j.jaip.2023.03.055. Epub 2023 Apr 23. No abstract available.

Reference Type: DERIVED

PMID: 37088376 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 33223095 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 32298853 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 19910033 (View on PubMed)

Other Identifiers

CIGE025AIA05

Identifier Type: -

Identifier Source: org_study_id