AzTrials
AzTrials

AsthmaPhase 2MUPPITS-2NCT03292588

A Trial of Mepolizumab Adjunctive Therapy for the Prevention of Asthma Exacerbations in Urban Children

A Phase 2 study testing Mepolizumab in people with Asthma, run by National Institute of Allergy and Infectious Diseases (NIAID) at 9 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 6 to 17 years
Conditions the study is for
Asthma
What is being tested?
Mepolizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
335 participants
Where
9 sitesUnited States
Study dates
7 Nov 2017to 20 Apr 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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);
Show all 13Show fewer
  • 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;
  • 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.

Who cannot join

  • 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
Show all 25Show fewer
  • --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,
  • May pose additional risks from participation in this study,
  • May interfere with the participant's ability to comply with study requirements, or
  • May impact the quality or interpretation of the data obtained from the study.
  • 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.

Trial locations and contacts

9 sites in 1 country.

9 sites in 1 country

United States9 sites
  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Ann and Robert Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Henry Ford Health System

    Detroit, Michigan, 48202

  • Columbia University Medical Center

    New York, New York, 10032

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Mechanisms Underlying Asthma Exacerbations Prevented and Persistent With Immune-Based Therapy: A Systems Approach Phase 2 (ICAC-30)

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.

Read the 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.

Conditions
Asthma
Being tested
Mepolizumab, Placebo
Keywords
urban setting, children and adolescents, double-masked, placebo-controlled randomized trial, asthma exacerbations (attacks), mepolizumab adjunctive therapy, asthma exacerbations prevention, difficult-to-control exacerbation-prone asthma
Medical subject headings
Asthma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MepolizumabBiological

    Also called Nucala®, anti-Interleukin 5 (IL5) antagonist monoclonal antibody

    Mepolizumab administered every 4 weeks by subcutaneous injection at a dose of:

    • 100 mg for participants ≥12 years of age and
    • 40 mg for participants ages 6 to 11 years and weighing ≥40 kg.

    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.

  • PlaceboDrug

    Also called Placebo for Mepolizumab

    Placebo administered every 4 weeks by subcutaneous injection at a dose of:

    • 100 mg for participants ≥12 years of age and
    • 40 mg for participants ages 6 to 11 years and weighing ≥40 kg.

    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.

Study groups in NCT03292588
Study groupRoleWhat they receive
MepolizumabExperimentalIntervention: Mepolizumab plus guidelines-based standard of care asthma treatment.
Receives: Biological: Mepolizumab
PlaceboPlacebo comparatorIntervention: Placebo for mepolizumab plus guidelines-based standard of care asthma treatment.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to 12 months

Secondary outcomes (12)
  1. 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.

    Measured: Week 12, 24, 36, 48, 52 after randomization

  2. 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.

    Measured: Week 56

  3. 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.

    Measured: Week 56

  4. 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.

    Measured: Weeks 12, 24, 36, 48, 52 after randomization

  5. 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.

    Measured: Weeks 12, 24, 36, 48, 52 after randomization

  6. 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

    Measured: Up to 12 months

  7. 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

    Measured: Up to 12 months

  8. 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).

    Measured: Up to 12 months

  9. 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.

    Measured: Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment)

  10. 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.

    Measured: Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment)

  11. 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.

    Measured: Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment)

  12. 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.

    Measured: Week 4 (Treatment Initiation) to Week 56 (Completion of Treatment)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Sep 2017
  2. Study start7 Nov 2017
  3. Primary completion20 Apr 2021
  4. Study completion20 Apr 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
335 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03292588
Sponsor's protocol ID
DAIT ICAC-30
Other IDs
UM1AI114271, NIAID CRMS ID#: 38189
Acronym
MUPPITS-2
Status last verified
Jun 2022
First submitted
20 Sep 2017
Last updated
20 Jul 2022
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Participant level data access and additional relevant materials will be made available upon completion of the trial.

Publications

  • 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.

    PubMed 40897727
  • 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.

    PubMed 40658400
  • 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.

    PubMed 35964610

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Mepolizumab in people with Asthma, run by National Institute of Allergy and Infectious Diseases (NIAID) at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 21 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Asthma Exacerbations During the Treatment Period (time frame: Up to 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03292588, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2022.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in