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Individualized Therapy For Asthma in Toddlers

Registration Number
NCT01606306
Lead Sponsor
Milton S. Hershey Medical Center
Brief Summary

The INFANT study will test whether, in preschool children 12-59 months of age with persistent asthma, the following Step 2 asthma therapies will provide similar degrees of asthma control:

1. Daily inhaled corticosteroid (ICS) treatment,

2. Daily leukotriene receptor antagonist (LTRA) treatment, and

3. As-needed ICS plus short-acting beta agonist (as-needed ICS/SABA) rescue treatment.

Detailed Description

INFANT is a double-blind, randomized clinical trial in which all participants will receive each of the three therapies for 16 weeks by means of a cross-over study design. INFANT aims to determine whether individual children respond better to one treatment than another and, if so, whether those children can be identified by phenotypic characteristics or selected biomarkers. In this regard the INFANT study is expected to address critical gaps in current asthma management guidelines. Ultimately, the findings from this study are expected to help clarify treatment modalities for this population of young preschool children who are extremely difficult to treat.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
300
Inclusion Criteria
  • 12-59 months of age.

  • If the child is not currently taking long-term asthma controller therapy (meaning that the child has taken no inhaled corticosteroid or leukotriene receptor antagonist medication whatsoever over the past 6 months), then one of the following criteria must be met:

    • Daytime asthma symptoms more than two days per week (average over the past 4 weeks),
    • At least one nighttime awakening from asthma (over the past 4 weeks),
    • Two or more asthma exacerbations requiring systemic corticosteroids in the previous 6 months,
    • Four or more wheezing episodes in the previous 12 months.
  • If the child is currently taking long-term asthma controller therapy (meaning that the child has taken daily or intermittent/as-needed inhaled corticosteroid or leukotriene receptor antagonist over the past 6 months), then one of the following criteria must be met:

    • Taking inhaled corticosteroid or leukotriene receptor antagonist for more than 3 months (or more than 90 days) out of the previous 6 months (or 180 days),
    • Daytime asthma symptoms more than two days per week (average over the past 4 weeks),
    • More than one nighttime awakening from asthma (over the past 4 weeks),
    • Two or more asthma exacerbations requiring systemic corticosteroids in the previous 12 months,
    • Four or more wheezing episodes in the previous 12 months.
  • Up to date with immunizations, including varicella (unless the subject has already had clinical varicella).

  • Willingness to provide informed consent by the child's parent or guardian.

Exclusion Criteria
  • Allergic reaction to the study medications or any component of the study drugs, including (but not limited to) urticaria, rash, angioedema, or hypotension following delivery,

  • Chronic medical disorders that could interfere with drug metabolism/excretion (for instance chronic hepatic, biliary, or renal disease),

  • Chronic medical disorders that may increase the risk of drug-related injury, including (but not limited to):

    • Osteogenesis imperfecta (increased risk of bone demineralization/fracture with corticosteroid therapy),
    • Crohn's disease, ulcerative colitis, juvenile rheumatoid arthritis, clotting disorders, or Factor deficiency (increased risk of bleeding with corticosteroid therapy),
    • G6PD deficiency (increased risk of hemolytic anemia with acetaminophen use),
    • Phenylketonuria (potential for aspartame exposure with study interventions),
    • Seizure disorder treated with anticonvulsants (risk of acetaminophen toxicity with carbamazepine), or
    • History of clotting disorders or Factor deficiency (increased risk of bleeding with corticosteroids),
  • Co-morbid disorders associated with wheezing including (but not limited to) immune deficiency disorders, cystic fibrosis, aspiration, clinically-relevant gastroesophageal reflux, tracheomalacia, congenital airway anomalies (clefts, fistulas, slings, rings), bronchiectasis, bronchopulmonary dysplasia, and/or history of premature birth before 35 weeks gestation,

  • Significant developmental delay/failure to thrive, defined as 5th percentile for height and/or weight or crossing of two major percentile lines during the last year for age and sex,

  • History of a near-fatal asthma exacerbation requiring intubation or assisted ventilation,

  • No primary medical caregiver (e.g., a nurse practitioner, physician assistant, physician, or group medical practice such as a hospital-based clinic) whom the subject can contact for primary medical care,

  • Three or more hospitalizations in the previous 12 months for wheezing or respiratory illnesses,

  • Treatment with 5 or more courses of systemic corticosteroids (oral, intramuscular or intravenous) in the past 6 months,

  • Current use of higher than step 2 NAEPP asthma guideline therapy

  • If receiving allergy shots, change in the dose within the past 3 months.

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
Crossover sequence 5daily fluticasone propionateas needed fluticasone propionate, followed by daily fluticasone propionate, followed by daily montelukast
Crossover sequence 1daily fluticasone propionatedaily fluticasone propionate, followed by daily montelukast, followed by as needed fluticasone propionate
Crossover sequence 1as-needed fluticasone propionatedaily fluticasone propionate, followed by daily montelukast, followed by as needed fluticasone propionate
Crossover sequence 2daily fluticasone propionatedaily fluticasone propionate, followed by as needed fluticasone propionate, followed by daily montelukast
Crossover sequence 4as-needed fluticasone propionatedaily montelukast, followed by daily fluticasone propionate, followed by as needed fluticasone propionate
Crossover sequence 5as-needed fluticasone propionateas needed fluticasone propionate, followed by daily fluticasone propionate, followed by daily montelukast
Crossover sequence 6daily fluticasone propionateas needed fluticasone propionate, followed by daily montelukast, followed by daily fluticasone propionate
Crossover sequence 2as-needed fluticasone propionatedaily fluticasone propionate, followed by as needed fluticasone propionate, followed by daily montelukast
Crossover sequence 3daily fluticasone propionatedaily montelukast, followed by as needed fluticasone propionate, followed by daily fluticasone propionate
Crossover sequence 6as-needed fluticasone propionateas needed fluticasone propionate, followed by daily montelukast, followed by daily fluticasone propionate
Crossover sequence 3as-needed fluticasone propionatedaily montelukast, followed by as needed fluticasone propionate, followed by daily fluticasone propionate
Crossover sequence 4daily fluticasone propionatedaily montelukast, followed by daily fluticasone propionate, followed by as needed fluticasone propionate
Crossover sequence 1Montelukastdaily fluticasone propionate, followed by daily montelukast, followed by as needed fluticasone propionate
Crossover sequence 2Montelukastdaily fluticasone propionate, followed by as needed fluticasone propionate, followed by daily montelukast
Crossover sequence 3Montelukastdaily montelukast, followed by as needed fluticasone propionate, followed by daily fluticasone propionate
Crossover sequence 4Montelukastdaily montelukast, followed by daily fluticasone propionate, followed by as needed fluticasone propionate
Crossover sequence 5Montelukastas needed fluticasone propionate, followed by daily fluticasone propionate, followed by daily montelukast
Crossover sequence 6Montelukastas needed fluticasone propionate, followed by daily montelukast, followed by daily fluticasone propionate
Primary Outcome Measures
NameTimeMethod
Differential Response to the Three Therapies Based on Fixed Threshold Criteria for the Following Asthma Control Measures: Use of Oral Prednisone for Acute Asthma Exacerbations and Asthma Control Days.The last 14 weeks of each 16-week treatment period

The primary outcome was differential response to the three therapies on the basis of fixed threshold criteria for the following asthma control measures, which encompassed domains of risk and impairment: the time from the start of the treatment period to an asthma exacerbation treated with systemic corticosteroids, and the annualized number of asthma control days (ACDs) from within that period. ACDs were defined as full calendar days without symptoms, rescue medication use, or unscheduled healthcare visits. Children were defined as differential responders if, first, the time to an asthma exacerbation was at least four weeks longer, or second, if the number of annualized ACDs was at least 31 days more for one treatment than another, in that order. If neither threshold was met, the participant was considered a non differential responder. Differential response was determined in children completing at least two treatment periods and at least 50% of the daily diary entries for each period.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (13)

St. Louis Children's Hospital

🇺🇸

St. Louis, Missouri, United States

University of Wisconsin-Madison

🇺🇸

Madison, Wisconsin, United States

University of Arizona College of Medicine

🇺🇸

Tucson, Arizona, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Children's Hospital & Research Center Oakland

🇺🇸

Oakland, California, United States

Children's Hospital of Pittsburgh of UPMC

🇺🇸

Pittsburgh, Pennsylvania, United States

UCSF Benioff Children's Hospital

🇺🇸

San Francisco, California, United States

Rush University Medical Center/Stroger Hospital

🇺🇸

Chicago, Illinois, United States

Children's Memorial Hospital

🇺🇸

Chicago, Illinois, United States

Children's Hospital, Boston

🇺🇸

Boston, Massachusetts, United States

Rainbow Babies and Children's Hospital, Case Western Reserve University

🇺🇸

Cleveland, Ohio, United States

National Jewish Health

🇺🇸

Denver, Colorado, United States

University of Virginia Health System

🇺🇸

Charlottesville, Virginia, United States

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