Mechanisms Underlying Asthma Exacerbations Prevented and Persistent With Immune-Based Therapy: A Systems Approach Phase 2 (ICAC-30)
Overview
- Phase
- Phase 2
- Status
- Completed
- Enrollment
- 335
- Locations
- 9
- Primary Endpoint
- Number of Asthma Exacerbations During the Treatment Period
Overview
Brief Summary
The purpose of this study is to see if treatment with a medication called Nucala® (mepolizumab), given along with standard asthma care, makes children less likely to have asthma attacks.
Detailed Description
Asthma is a growing problem, especially in children. It causes frequent wheezing, shortness of breath, chest tightness, and cough. Asthma attacks, or exacerbations, are problems for children with asthma.
The purpose of this study is to see if treatment with a medication called mepolizumab (Nucala®), given along with standard asthma care, makes children less likely to have asthma attacks. Mepolizumab is a new drug that is approved by the Food and Drug Administration (FDA) for use in children with asthma who are aged 12 years and older. Mepolizumab is given by injection. It is being studied by other researchers in children aged 6-11 years.
All participants will be prescribed standard asthma medications by a clinician who is trained in asthma care. Medications will include controller medications, a rescue medication, and a medication for severe asthma attacks (prednisone). The amount of medication that participants receive may be increased or decreased during the study based on their symptoms and breathing test results. Study clinicians will treat all participants according to the same guidelines. These treatment guidelines are based on recommendations from a group of national experts in asthma. This study has been designed this way so that all participants will have safe and effective standard asthma care.
In order to enroll in this study, participants must be willing to have their asthma managed by the study clinician during the entire study period. Participants must also be willing to bring study medications to all study visits.
This study will include up to 20 study visits. Participant involvement in the study will endure for approximately 1 year.
During the treatment period, participants will be placed in one of two treatment groups:
- Mepolizumab injection and guidelines-based asthma care or
- Placebo injection and guidelines-based asthma care.
Participants will not be able to choose which group they are assigned. This assignment is random and by chance, much like flipping a coin. Participants will not know if they are receiving mepolizumab or placebo. Investigators will compare the study results between the participants of each group.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Eligibility Criteria
- Ages
- 6 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Study applicant(s) that fulfill all of the inclusion criteria and none of the exclusion criteria are eligible for the study-
- •Participant and/or parent guardian must be able to understand and provide informed consent and age-appropriate assent;
- •Must have a primary place of residence in one of the pre-selected recruitment census tracts as outlined in the study's Manual of Procedures (MOP);
- •Has had a diagnosis of asthma made \>1 year prior to recruitment;
- •-Those who received an asthma diagnosis by a clinician ≤1 year prior to recruitment must report that their respiratory symptoms were present for more than 1 year prior to recruitment.
- •Has had ≥2 asthma exacerbations in the prior year (defined as a requirement for systemic corticosteroids and/or hospitalization);
- •At Visit 0 (Screening), has the following requirement for asthma controller medication:
- •For those ages 6 to 11 years, treatments with at least fluticasone 250 mcg dry powder inhaler (DPI) one puff twice daily or its equivalent and,
- •For those ≥12 years of age, treatment with at least Advair 250/50 mcg dry powder inhaler (DPI), one puff twice daily or its equivalent.
- •Has peripheral blood eosinophils ≥150 cells/µl obtained at Visit 0 (Screening) or in another Inner-City Asthma Consortium (ICAC) clinical research study within 6 months;
Exclusion Criteria
- •Individual(s) who meets any of the following criteria are not eligible for enrollment or randomization-
- •Is not able or willing to give written informed consent or comply with the study protocol;
- •Has concurrent (existing) medical problems that would require systemic corticosteroids or other immunomodulator treatments during the study;
- •Is currently receiving immunotherapy;
- •Is currently receiving treatment with omalizumab or has had omalizumab treatment within 6 months prior to planned participant randomization to treatment assignment;
- •Is currently requiring greater than fluticasone 500 mcg administered twice daily plus a long-acting beta agonist (LABA) one puff twice daily or its equivalent, and/or
- •-Individuals using oral corticosteroids daily or every other day for more than 14 days at the time of Visit 0 (Screening).
- •Is currently pregnant or lactating, or plans to become pregnant during the time of study participation
- •-Note: Females of child-bearing potential (post-menarche) must be abstinent or use a medically acceptable birth control method throughout the study (e.g. oral subcutaneous, mechanical, or surgical contraception).
- •Has a known, pre-existing clinically important lung condition other than asthma;
Arms & Interventions
Mepolizumab
Intervention: Mepolizumab plus guidelines-based standard of care asthma treatment.
Intervention: Mepolizumab (Biological)
Placebo
Intervention: Placebo for mepolizumab plus guidelines-based standard of care asthma treatment.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Number of Asthma Exacerbations During the Treatment Period
Time Frame: Up to 12 months
Exacerbations were defined as a prescription of a course of systemic corticosteroids by a clinician, initiation of a course of systemic corticosteroids by a participant, or as a hospitalization for asthma. If a participant initiated and completed a course of systemic corticosteroids without clinician involvement, this course was counted only if the study clinician agreed the treatment was warranted, and it met the following dosage: the course for prednisone, prednisolone, or methylprednisolone was at least 20 mg daily dose for 3 of 5 consecutive days. The course for dexamethasone was at least a 10 mg single daily dose. If a corticosteroid burst for the treatment of an asthma exacerbation was prescribed by a non-ICAC clinician, it was counted regardless of dose.
Secondary Outcomes
- Number of Reported Adverse Events (AEs), Including Their Severity(Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment))
- Time to First Asthma Exacerbation(Up to 12 months)
- Composite Asthma Severity Index (CASI)(Week 12, 24, 36, 48, 52 after randomization)
- Participant Quality of Life Measured Using the Physician Global Assessment Tool(Week 56)
- Participant Quality of Life Measured Using the Patient Global Assessment, at Visit 14(Week 56)
- Lung Function as Assessed by Impulse Oscillometry(Weeks 12, 24, 36, 48, 52 after randomization)
- Rate of Exacerbations (Mepolizumab vs. Placebo) During the Treatment Period for Participants Who Did Not Fit the FDA-approved Dosing Table for Omalizumab Therapy.(Up to 12 months)
- Lung Function as Assessed by Spirometry(Weeks 12, 24, 36, 48, 52 after randomization)
- Rate of Exacerbations (Mepolizumab vs. Placebo) During the Treatment Period for Participants Who Fit the FDA-approved Dosing Table.(Up to 12 months)
- Number of Reported Adverse Events (AEs), Including Their Treatment Relatedness(Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment))
- Number of Reported Serious Adverse Events (SAEs) Inclusive of Severity. Please Refer to the Adverse Event Tables for Specifics.(Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment))
- Number of Reported Serious Adverse Events (SAEs) Inclusive of Treatment Relatedness. Please Refer to the Adverse Event Tables for Specifics.(Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment))