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New combo treatment shows promise for kids with rare bone marrow failure

NCT ID NCT07415083

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study reviewed medical records of 13 Korean children with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Researchers wanted to see if adding eltrombopag to standard immunosuppressive therapy was safe and effective. Over 6 months, they tracked side effects and blood cell recovery. The goal was to improve blood counts without causing serious harm.

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.

Participants

13 people

The number who actually took part.

Started

Jun 2023

Finished

Feb 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.

Who is studied

Pediatric patients with sAA who initiated eltrombopag and IST treatment in Korea.

Ages

2 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: 1. Male or female pediatric patients aged ≥2 years and \<18 years at the time of initiating eltrombopag and IST treatment. 2. Subjects diagnosed with sAA and treated with eltrombopag in combination with IST as first-line therapy, based on the physician's decision in accordance with locally approved prescribing information at the time of data collection. 3. Subjects whose analytical index date (start date of eltrombopag administration) was at least 6 months prior to the data extraction date. Exclusion criteria: None.

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Conditions

The condition(s) this trial relates to.

aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Aplastic anemia

Contacts and locations

Locations

  • Novartis

    East Hanover, New Jersey, 07963, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.