NCT07415083

Brief Summary

The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia (sAA) in Korea. The study used data over a 6-month period extracted from patients' medical charts (electronic medical records) starting from the initiation of eltrombopag treatment.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jun 2023

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 21, 2023

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 25, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 25, 2025

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

February 11, 2026

Completed
6 days until next milestone

First Posted

Study publicly available on registry

February 17, 2026

Completed
Last Updated

February 19, 2026

Status Verified

February 1, 2026

Enrollment Period

1.7 years

First QC Date

February 11, 2026

Last Update Submit

February 16, 2026

Conditions

Aplastic Anemia

Keywords

Severe Aplastic AnemiaEltrombopagRetrospective chart reviewSafety

Outcome Measures

Primary Outcomes (1)

  • Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Incidence of all AEs/adverse drug reactions (ADRs), SAEs/serious adverse drug reactions (SADRs), and unexpected AEs/ADRs.

    Up to 6 months

Secondary Outcomes (1)

  • Overall Response Rate (ORR)

    6 months

Study Arms (1)

Eltrombopag and IST Treatment Cohort

Pediatric patients with sAA who received first-line eltrombopag in combination with IST.

Eligibility Criteria

Age2 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • Male or female pediatric patients aged ≥2 years and \<18 years at the time of initiating eltrombopag and IST treatment.
  • 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.
  • Subjects whose analytical index date (start date of eltrombopag administration) was at least 6 months prior to the data extraction date.

You may not qualify if:

  • None.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Novartis

East Hanover, New Jersey, 07963, United States

Location

Related Links

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 11, 2026

First Posted

February 17, 2026

Study Start

June 21, 2023

Primary Completion

February 25, 2025

Study Completion

February 25, 2025

Last Updated

February 19, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)