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New hope for kids with rare blood disorder: drug may cut flares and steroid use

NCT ID NCT04965636

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

16 people

The number who actually took part.

Started

Jul 2022

Finished

Oct 2025

Lead sponsor

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

6 to 17 years

Sex

Anyone

Healthy volunteers

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

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

  • GSK Investigational Site

    Rochester, Minnesota, 55905, United States

  • GSK Investigational Site

    Cincinnati, Ohio, 45229, United States

  • GSK Investigational Site

    Cleveland, Ohio, 44106, United States

  • GSK Investigational Site

    Charleston, South Carolina, 29425, United States

  • GSK Investigational Site

    Buenos Aires, 1888, Argentina

  • GSK Investigational Site

    Buenos Aires, C1028AAP, Argentina

  • GSK Investigational Site

    Quilmes, 1878, Argentina

  • GSK Investigational Site

    São Paulo, 05410-002, Brazil

  • GSK Investigational Site

    Sorocaba, 18040-425, Brazil

  • GSK Investigational Site

    Petah Tikva, 49202, Israel

  • GSK Investigational Site

    Rotterdam, 3015 CE, Netherlands

  • GSK Investigational Site

    Ankara, 6230, Turkey (Türkiye)

  • GSK Investigational Site

    Izmir, 35100, Turkey (Türkiye)

  • GSK Investigational Site

    Kayseri, 38039, Turkey (Türkiye)

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