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| ID | Type | Description | Link |
|---|---|---|---|
| UM1AI114271 | U.S. NIH Grant/Contract | View source | |
| NIAID CRMS ID#: 38189 | Other Identifier | DAIT NIAID |
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| Name | Class |
|---|---|
| Inner-City Asthma Consortium | NETWORK |
| GlaxoSmithKline | INDUSTRY |
| Rho Federal Systems Division, Inc. | INDUSTRY |
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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.
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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mepolizumab | Experimental | Intervention: Mepolizumab plus guidelines-based standard of care asthma treatment. |
|
| Placebo | Placebo Comparator | Intervention: Placebo for mepolizumab plus guidelines-based standard of care asthma treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mepolizumab | Biological | Mepolizumab administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Asthma Exacerbations During the Treatment Period | 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. | Up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Composite Asthma Severity Index (CASI) | Composite Asthma Severity Index (CASI) scores included 5 domains: day symptoms and albuterol use, night symptoms and albuterol use, controller treatment, lung function measures, and exacerbations. The minimum composite score was 0 while the maximum was 20. The higher the score the more allergy symptoms a subject has. | Week 12, 24, 36, 48, 52 after randomization |
| Measure | Description | Time Frame |
|---|---|---|
| EXPLORATORY: Time to First Respiratory Virus-Induced Exacerbation | As measured by an exacerbation associated with a respiratory virus detected using nasal mucus samples obtained at the time of an exacerbation. | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
| EXPLORATORY:Number of Respiratory Virus-Induced Exacerbations |
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:
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;
Is able to perform spirometry at randomization (Visit for treatment assignment);
Has documentation of current medical insurance with prescription coverage at randomization; and
Has had varicella or the varicella vaccination.
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;
Has a current malignancy or previous history of cancer in remission for less than 12 months prior to randomization;
Has known, pre-existing, unstable liver disease;
Is a current smoker or has a smoking history of 10 or more pack years;
Has a known immunodeficiency disease;
Has other conditions that could lead to elevated eosinophils such as hypereosinophilic syndromes, including eosinophilic granulomatosis with polyangiitis;
Has a known, active pre-existing parasitic infestation or is undergoing treatment for a parasitic infestation
--Note: Once the individual has been successfully treated, the interested study applicant may be reevaluated for study eligibility.
Positive for use of investigational drugs within 4 weeks of randomization;
Has a past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the study clinician,
In the event that the study applicant will not allow the study clinician, an asthma specialist, to manage their disease for the duration of the study or who are not willing to change their asthma medications to follow the protocol;
Has a known history of allergic reaction to previous biologic therapy for asthma; or
Has had a life threatening asthma exacerbation in the last 2 years requiring intubation, mechanical ventilation or resulting in a hypoxic seizure.
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| Name | Affiliation | Role |
|---|---|---|
| Daniel J Jackson, MD | University of Wisconsin, Madison | Study Chair |
| William W Busse, MD | University of Wisconsin, Madison | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States | ||
| Children's National Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40897727 | Derived | Gaberino CL, Segnitz RM, Dill-McFarland KA, Bacharier LB, Calatroni A, Gill MA, Stokes J, Liu AH, Cohen RT, Kumar R, Lang A, Khurana Hershey GK, Sherenian MG, Zoratti EM, Teach SJ, Kattan M, Becker PM, Togias A, Busse WW, Jackson DJ, Altman MC. Mepolizumab alters gene regulatory networks of nasal airway type-2 and epithelial inflammation in urban children with asthma. Nat Commun. 2025 Sep 2;16(1):8191. doi: 10.1038/s41467-025-63629-2. | |
| 40658400 | Derived |
| Label | URL |
|---|---|
| National Institute of Allergy and Infectious Diseases (NIAID) website | View source |
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Participant level data access and additional relevant materials will be made available upon completion of the trial.
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After completion of the trial, within 24 months status post database lock.
Registration is available for the Immunology Database and Analysis Portal (ImmPort) at: https://www.immport.org/registration. Submit a rationale for the purpose of requesting study data access.
ImmPort is a long-term archive of clinical and mechanistic data, a National Institute of Allergy and Infectious Diseases Division of Allergy, Immunology and Transplantation (NIAID DAIT)-funded data repository. This archive is in support of the NIH mission to share data with the public. Data shared through ImmPort is provided by NIH-funded programs, other research organizations and individual scientists, ensuring these discoveries will be the foundation of future research.
335 people were enrolled in the study (signed a consent and were assigned a unique participant number). However, 45 participants dropped out before receiving treatment bringing the total to 290 participants who started treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Mepolizumab | Intervention: Mepolizumab plus guidelines-based standard of care asthma treatment. Mepolizumab: Mepolizumab administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 1, 2021 |
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|
|
| Placebo | Drug | Placebo administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
|
|
| Participant Quality of Life Measured Using the Physician Global Assessment Tool | The Physician Global Assessment Tool was used to assess the quality of life of the subjects during treatment. The questionnaire is one question that asks the physician to evaluate how the participant's quality of life changed over the course of treatment. There are seven possible options ranging from significantly worse to significantly improved. | Week 56 |
| Participant Quality of Life Measured Using the Patient Global Assessment, at Visit 14 | The Patient Global Assessment Tool was used to assess the quality of life of the subjects during treatment. The questionnaire is one question that asks the participant to evaluate how their quality of life changed over the course of treatment. There are seven possible options ranging from significantly worse to significantly improved. | Week 56 |
| Lung Function as Assessed by Spirometry | A generalized mixed model was used to analyze spirometry parameter at each visit where the lung function was collected. The ratio of the forced expiratory volume to the forced vital capacity of the lungs (FEV1/FVC) is the outcome that measured lung function. | Weeks 12, 24, 36, 48, 52 after randomization |
| Lung Function as Assessed by Impulse Oscillometry | A generalized mixed model was used to analyze impulse oscillometry parameter at each visit where the lung function was collected. The Percent Predicted FEV1 (%) is the outcome that measured lung function. | 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. | Looking at the rate of exacerbations similarly to the primary endpoint. This outcome measure also took into consideration FDA- approved dosing of omalizumab. The FDA-approved dosing table is based off of age, weight and pre-treatment Serum IGE | Up to 12 months |
| Rate of Exacerbations (Mepolizumab vs. Placebo) During the Treatment Period for Participants Who Fit the FDA-approved Dosing Table. | Looking at the rate of exacerbations similarly to the primary endpoint. This outcome measure also took into consideration FDA- approved dosing of omalizumab. The FDA-approved dosing table is based off of age, weight and pre-treatment Serum IGE | Up to 12 months |
| Time to First Asthma Exacerbation | A Cox PH model was also used to model the time to first asthma exacerbation during the treatment period. The Cox PH model included treatment arm as the primary exposure but was also adjusted for study site, number of exacerbations in year prior to study (2 or 3+), peripheral blood eosinophils (above or below 400 cells/μl), BMI (above or below 95th percentile for age) and total serum IgE (above or below 540 kUA/L). | Up to 12 months |
| Number of Reported Adverse Events (AEs), Including Their Severity | The number of AEs by severity was used to assess safety. Please refer to the Adverse Event tables for specifics. | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
| Number of Reported Adverse Events (AEs), Including Their Treatment Relatedness | The number of AEs by relationship to study drug was used to assess safety. 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 Severity. Please Refer to the Adverse Event Tables for Specifics. | The number of SAEs by severity and relationship to study drug was used to assess safety. | 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. | The number of SAEs by relationship to study drug was used to assess safety. | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
Measured by an exacerbation associated with a respiratory virus detected using nasal mucus samples obtained at the time of an exacerbation. |
| Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
| EXPLORATORY:Childhood Asthma Control Test (ACT)/c-ACT | A validated tool to assess overall asthma control (over the last 4 weeks) in participants. | Visits (V) 4 (Week 4 Treatment Initiation) , V4 (Week 16), V7 (Week 28), V10 (Week 40), V13 (Week 52) and V14 (Week 56, Completion of Treatment) |
| EXPLORATORY:Maximum Number of Asthma Symptom Days | Defined as the highest value among the following variables over a two-week period:
| Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
| EXPLORATORY:Bronchodilator Responsiveness | A measure to determine whether mepolizumab improves pulmonary outcomes. | Baseline (prior to treatment initiation), Week 56 (Completion of Treatment) |
| EXPLORATORY: Gene Expression in Nasal Lavage Samples | Whole genome transcriptomics of nasal lavage samples to identify inflammatory pathways affected by mepolizumab. | Visits (V) 1 (Week 4 Treatment Initiation) , V3(Week 12), and V14 (Week 56, Completion of Treatment) |
| EXPLORATORY: Gene Expression in Whole Blood RNA Samples | Whole genome transcriptomics of whole blood RNA samples to identify inflammatory pathways affected by mepolizumab.. | Visits (V) 1 (Week 4 Treatment Initiation) , V3(Week 12), and V14 (Week 56, Completion of Treatment) |
| EXPLORATORY:Levels of Antibody to Mepolizumab | An assay for detection/measurement of levels of antibody to mepolizumab. Analysis will include participants randomized to mepolizumab. | Visit (V) 1 (Week 4, prior to treatment initiation), V3 (Week 12), and Visit 14 (Week 56, Completion of Treatment) |
| EXPLORATORY:Other Potential Biomarkers Not Specified to Date | Plasma, nasal samples, RNA and DNA will be banked for possible future study of potential biomarkers associated with asthma and asthma exacerbations. | Visit (V) 1 (Week 4, prior to treatment initiation), V3 (Week 12), and Visit 14 (Week 56, Completion of Treatment) |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Ann and Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Altman MC, Janczyk T, Murphy RC, Jayavelu ND, Calatroni A, Kattan M, Gill MA, Stokes J, Liu AH, Khurana Hershey GK, Sherenian M, Kumar R, Robison RG, Gruchalla RS, O'Connor GT, Zoratti EM, Teach SJ, Lynch SV, Dill-McFarland KA, Becker PM, Togias A, Gern JE, Bacharier LB, Busse WW, Jackson DJ; Inner City Asthma Consortium and Childhood Asthma in Urban Settings Network. Inflammatory Pathways in Residual Asthma Exacerbations Among Mepolizumab-Treated Urban Children: A Secondary Analysis of a Randomized Clinical Trial. JAMA Pediatr. 2025 Sep 1;179(9):957-970. doi: 10.1001/jamapediatrics.2025.2044. |
| 35964610 | Derived | Jackson DJ, Bacharier LB, Gergen PJ, Gagalis L, Calatroni A, Wellford S, Gill MA, Stokes J, Liu AH, Gruchalla RS, Cohen RT, Makhija M, Khurana Hershey GK, O'Connor GT, Pongracic JA, Sherenian MG, Rivera-Spoljaric K, Zoratti EM, Teach SJ, Kattan M, Dutmer CM, Kim H, Lamm C, Sheehan WJ, Segnitz RM, Dill-McFarland KA, Visness CM, Becker PM, Gern JE, Sorkness CA, Busse WW, Altman MC; US National Institute of Allergy and Infectious Disease's Inner City Asthma Consortium. Mepolizumab for urban children with exacerbation-prone eosinophilic asthma in the USA (MUPPITS-2): a randomised, double-blind, placebo-controlled, parallel-group trial. Lancet. 2022 Aug 13;400(10351):502-511. doi: 10.1016/S0140-6736(22)01198-9. |
| Division of Allergy, Immunology, and Transplantation (DAIT) website | View source |
| FG001 | Placebo | Intervention: Placebo for mepolizumab plus guidelines-based standard of care asthma treatment. Placebo: Placebo administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
| COMPLETED |
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| NOT COMPLETED |
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Intent-to-treat population with available data
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| ID | Title | Description |
|---|---|---|
| BG000 | Mepolizumab | Intervention: Mepolizumab plus guidelines-based standard of care asthma treatment. Mepolizumab: Mepolizumab administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
| BG001 | Placebo | Intervention: Placebo for mepolizumab plus guidelines-based standard of care asthma treatment. Placebo: Placebo administered every 4 weeks by subcutaneous injection at a dose of:
Note: Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in the study under previous versions of the protocol and were initially assigned a 100 mg dose will have their dose reduced to 40 mg. Participants 11 years of age will increase to the 100 mg dose if they become age 12 years during the study. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Number of Asthma Exacerbations During the Treatment Period | 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. | All participants who were randomized and received at least one dose of study treatment | Posted | Mean | Standard Error | Exacerbations during treatment | Up to 12 months |
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| Secondary | Composite Asthma Severity Index (CASI) | Composite Asthma Severity Index (CASI) scores included 5 domains: day symptoms and albuterol use, night symptoms and albuterol use, controller treatment, lung function measures, and exacerbations. The minimum composite score was 0 while the maximum was 20. The higher the score the more allergy symptoms a subject has. | All participants who were randomized and received at least one dose of study treatment | Posted | Least Squares Mean | Standard Error | Composite Asthma Severity Index | Week 12, 24, 36, 48, 52 after randomization |
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| Secondary | Participant Quality of Life Measured Using the Physician Global Assessment Tool | The Physician Global Assessment Tool was used to assess the quality of life of the subjects during treatment. The questionnaire is one question that asks the physician to evaluate how the participant's quality of life changed over the course of treatment. There are seven possible options ranging from significantly worse to significantly improved. | All participants who were randomized and received at least one dose of study treatment and completed the quality of life questionnaire at visit 14. | Posted | Count of Participants | Participants | Week 56 |
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| Secondary | Participant Quality of Life Measured Using the Patient Global Assessment, at Visit 14 | The Patient Global Assessment Tool was used to assess the quality of life of the subjects during treatment. The questionnaire is one question that asks the participant to evaluate how their quality of life changed over the course of treatment. There are seven possible options ranging from significantly worse to significantly improved. | All participants who were randomized and received at least one dose of study treatment and completed the quality of life questionnaire at visit 14. | Posted | Count of Participants | Participants | Week 56 |
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| Secondary | Lung Function as Assessed by Spirometry | A generalized mixed model was used to analyze spirometry parameter at each visit where the lung function was collected. The ratio of the forced expiratory volume to the forced vital capacity of the lungs (FEV1/FVC) is the outcome that measured lung function. | All participants who were randomized and received at least one dose of study treatment | Posted | Least Squares Mean | Standard Error | FEV1/FVC | Weeks 12, 24, 36, 48, 52 after randomization |
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| Secondary | Lung Function as Assessed by Impulse Oscillometry | A generalized mixed model was used to analyze impulse oscillometry parameter at each visit where the lung function was collected. The Percent Predicted FEV1 (%) is the outcome that measured lung function. | Posted | Least Squares Mean | Standard Error | Percent Predicted FEV1 (%) | Weeks 12, 24, 36, 48, 52 after randomization |
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| Secondary | Rate of Exacerbations (Mepolizumab vs. Placebo) During the Treatment Period for Participants Who Did Not Fit the FDA-approved Dosing Table for Omalizumab Therapy. | Looking at the rate of exacerbations similarly to the primary endpoint. This outcome measure also took into consideration FDA- approved dosing of omalizumab. The FDA-approved dosing table is based off of age, weight and pre-treatment Serum IGE | Posted | Mean | Standard Error | Exacerbations during treatment | Up to 12 months |
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| Secondary | Rate of Exacerbations (Mepolizumab vs. Placebo) During the Treatment Period for Participants Who Fit the FDA-approved Dosing Table. | Looking at the rate of exacerbations similarly to the primary endpoint. This outcome measure also took into consideration FDA- approved dosing of omalizumab. The FDA-approved dosing table is based off of age, weight and pre-treatment Serum IGE | Posted | Mean | Standard Error | Exacerbations during treatment | Up to 12 months |
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| Secondary | Time to First Asthma Exacerbation | A Cox PH model was also used to model the time to first asthma exacerbation during the treatment period. The Cox PH model included treatment arm as the primary exposure but was also adjusted for study site, number of exacerbations in year prior to study (2 or 3+), peripheral blood eosinophils (above or below 400 cells/μl), BMI (above or below 95th percentile for age) and total serum IgE (above or below 540 kUA/L). | All participants who were randomized and received at least one dose of study treatment | Posted | Median | Full Range | Days | Up to 12 months |
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| Secondary | Number of Reported Adverse Events (AEs), Including Their Severity | The number of AEs by severity was used to assess safety. Please refer to the Adverse Event tables for specifics. | Number of Subjects with at Least One AE by severity | Posted | Count of Participants | Participants | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
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| Secondary | Number of Reported Adverse Events (AEs), Including Their Treatment Relatedness | The number of AEs by relationship to study drug was used to assess safety. Please refer to the Adverse Event tables for specifics. | Number of Subjects with any study procedures related AEs | Posted | Count of Participants | Participants | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
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| Secondary | Number of Reported Serious Adverse Events (SAEs) Inclusive of Severity. Please Refer to the Adverse Event Tables for Specifics. | The number of SAEs by severity and relationship to study drug was used to assess safety. | Number of subjects with Serious Adverse Events | Posted | Count of Participants | Participants | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
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| Other Pre-specified | EXPLORATORY: Time to First Respiratory Virus-Induced Exacerbation | As measured by an exacerbation associated with a respiratory virus detected using nasal mucus samples obtained at the time of an exacerbation. | Not Posted | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Number of Respiratory Virus-Induced Exacerbations | Measured by an exacerbation associated with a respiratory virus detected using nasal mucus samples obtained at the time of an exacerbation. | Not Posted | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Childhood Asthma Control Test (ACT)/c-ACT | A validated tool to assess overall asthma control (over the last 4 weeks) in participants. | Not Posted | Visits (V) 4 (Week 4 Treatment Initiation) , V4 (Week 16), V7 (Week 28), V10 (Week 40), V13 (Week 52) and V14 (Week 56, Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Maximum Number of Asthma Symptom Days | Defined as the highest value among the following variables over a two-week period:
| Not Posted | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Bronchodilator Responsiveness | A measure to determine whether mepolizumab improves pulmonary outcomes. | Not Posted | Baseline (prior to treatment initiation), Week 56 (Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY: Gene Expression in Nasal Lavage Samples | Whole genome transcriptomics of nasal lavage samples to identify inflammatory pathways affected by mepolizumab. | Not Posted | Visits (V) 1 (Week 4 Treatment Initiation) , V3(Week 12), and V14 (Week 56, Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY: Gene Expression in Whole Blood RNA Samples | Whole genome transcriptomics of whole blood RNA samples to identify inflammatory pathways affected by mepolizumab.. | Not Posted | Visits (V) 1 (Week 4 Treatment Initiation) , V3(Week 12), and V14 (Week 56, Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Levels of Antibody to Mepolizumab | An assay for detection/measurement of levels of antibody to mepolizumab. Analysis will include participants randomized to mepolizumab. | Not Posted | Visit (V) 1 (Week 4, prior to treatment initiation), V3 (Week 12), and Visit 14 (Week 56, Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | EXPLORATORY:Other Potential Biomarkers Not Specified to Date | Plasma, nasal samples, RNA and DNA will be banked for possible future study of potential biomarkers associated with asthma and asthma exacerbations. | Not Posted | Visit (V) 1 (Week 4, prior to treatment initiation), V3 (Week 12), and Visit 14 (Week 56, Completion of Treatment) | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Reported Serious Adverse Events (SAEs) Inclusive of Treatment Relatedness. Please Refer to the Adverse Event Tables for Specifics. | The number of SAEs by relationship to study drug was used to assess safety. | Number of subjects with any study procedures related Serious Adverse Events | Posted | Count of Participants | Participants | Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment) |
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Adverse events were collected from the time of consent until 1 year and the participant completes study participation or until 30 days after he/she prematurely withdraws (without withdrawing consent) or is withdrawn from the study.
The safety population was used for reporting adverse events, which was defined as: participants who were randomized and received at least one dose of study treatment. Participants were analyzed according to the medication they actually received, regardless of the treatment arm to which they were randomized. Non-treatment emergent adverse events were summarized in all participants who were enrolled, while treatment emergent adverse events were summarized in the safety sample
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Mepolizumab | Mepolizumab (anti-IL-5 antagonist monoclonal antibody) by subcutaneous injection every 4 weeks at the following doses: Age 12 years and above-100 mg, Age 6-11 years-40mg, Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in Protocol ICAC-30 under the previous versions of the protocol and initially assigned a 100 mg dose will have their dose reduced to 40 mg | 1 | 146 | 4 | 146 | 53 | 146 |
| EG001 | Placebo | Placebo by subcutaneous injection every 4 weeks at the following doses: Age 12 years and above-100 mg, Age 6-11 years-40mg, Participants 6 to 11 years of age and weighing ≥40 kg who were enrolled in Protocol ICAC-30 under the previous versions of the protocol and initially assigned a 100 mg dose will have their dose reduced to 40 mg | 0 | 144 | 2 | 144 | 42 | 144 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| All-Cause Mortality | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Chest pain | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Major depression | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Injection site reaction | General disorders | MedDRA 18.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Clinical Research Operations Program | DAIT/NIAID | 301-594-7669 | DAITClinicalTrialsGov@niaid.nih.gov |
| Apr 19, 2022 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D001249 | Asthma |
| ID | Term |
|---|---|
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
| D008173 | Lung Diseases, Obstructive |
| D008171 | Lung Diseases |
| D012130 | Respiratory Hypersensitivity |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C434107 | mepolizumab |
| D015848 | Interleukin-5 |
| ID | Term |
|---|---|
| D007378 | Interleukins |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
Not provided
Not provided
| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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