New hope for kids with rare blood disorder: drug may cut flares and steroid use
NCT ID NCT04965636
First seen Jun 25, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This study tested the drug mepolizumab in 16 children aged 6 to 17 with hypereosinophilic syndrome (HES), a rare condition causing too many eosinophils (a type of white blood cell). The goal was to see if mepolizumab could reduce disease flares and lower the need for steroids over 52 weeks. All participants received the drug alongside their usual care, and researchers tracked how many flares occurred and changes in steroid dose.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- mepolizumab
- What this could lead to
- If it works, this could provide a new treatment option to control hypereosinophilic syndrome in children and reduce their need for steroids.
- What could go wrong
- This is a small, single-arm study with only 16 participants, so results may not apply to all children. It also does not compare mepolizumab against a placebo or other treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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16 people
The number who actually took part.
- Started
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Jul 2022
- Finished
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Oct 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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6 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Participant must be aged 6 to 17 years inclusive, at Screening (Visit 1). * Participants who have been diagnosed with HES for at least 6 months prior to enrolment (Visit 2). * A history of 2 or more HES flares within the past 12 months prior to Screening (Visit 1). * Participants must have blood eosinophil count \>=1000 cells per microliter (/mcL) present at Screening. * Participants must be on a stable dose of HES therapy for the 4 weeks prior to the first dose of mepolizumab (Visit 2) * Male and/or female * Signed written informed consent Exclusion Criteria: * Life-threatening HES or life-threatening HES co-morbidities * Other concurrent medical conditions that may affect the participant's safety * Eosinophilia of unknown significance * Fusion tyrosine kinase gene translocation \[FIP1L1- Platelet-derived Growth Factor Receptor (PDGFRα) (F/P)\] positivity * Clinical diagnosis of eosinophilic granulomatosis with polyangiitis (EGPA) * Participants with chronic or ongoing active infections requiring systemic treatment, as well as participants who have experienced clinically significant infections due to viruses, bacteria, and fungi within 4 weeks prior to enrolment (Visit 2) * Participants with a pre-existing parasitic infestation within 6 months prior to enrolment (Visit 2) * Participants with a known immunodeficiency (e.g. Human immunodeficiency virus \[HIV\]), other than that explained by the use of OCS or other therapy taken for HES * Participants with documented history of any clinically significant cardiac damage prior to Screening (Visit 1) that, in the opinion of the investigator, would impact the participant's participation during the study * Participants with a history of or current lymphoma, Participants with current malignancy or previous history of cancer in remission for less than 12 months prior to Screening (Visit 1) * Participants who are not responsive to OCS based on clinical response or blood eosinophil counts. * Participants who have previously received mepolizumab in the 4 months prior to enrolment (Visit 2) * Participants receiving non-oral systemic corticosteroids in the 4-week period prior to enrolment (Visit 2). * Participants who have received any other monoclonal antibodies within 30 days or 5 half-lives, whichever is longer, of enrolment (Visit 2). * Participants who have received treatment with an investigational agent (biologic or non-biologic) within the past 30 days or 5 drug half-lives, whichever is longer, prior to enrolment (Visit 2). * Use of candidate Coronavirus disease 2019 (COVID-19) vaccines that have not received limited, accelerated, or full authorization/approval, and are only in use as part of a clinical trial. * Participants who are currently participating in any other interventional clinical study * Participants with any history of hypersensitivity to any monoclonal antibody (including mepolizumab). * Evidence of clinically significant abnormality in the hematological, biochemical, or urinalysis screen from the sample collected at Screening (Visit 1), that could put the participant's safety at risk by participating in the study, as judged by the investigator
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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GSK Investigational Site
Rochester, Minnesota, 55905, United States
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GSK Investigational Site
Cincinnati, Ohio, 45229, United States
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GSK Investigational Site
Cleveland, Ohio, 44106, United States
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GSK Investigational Site
Charleston, South Carolina, 29425, United States
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GSK Investigational Site
Buenos Aires, 1888, Argentina
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GSK Investigational Site
Buenos Aires, C1028AAP, Argentina
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GSK Investigational Site
Quilmes, 1878, Argentina
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GSK Investigational Site
São Paulo, 05410-002, Brazil
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GSK Investigational Site
Sorocaba, 18040-425, Brazil
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GSK Investigational Site
Petah Tikva, 49202, Israel
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GSK Investigational Site
Rotterdam, 3015 CE, Netherlands
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GSK Investigational Site
Ankara, 6230, Turkey (Türkiye)
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GSK Investigational Site
Izmir, 35100, Turkey (Türkiye)
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GSK Investigational Site
Kayseri, 38039, Turkey (Türkiye)
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