Aplastic AnemiaNCT07415083
A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
An observational study of Aplastic Anemia, run by Novartis Pharmaceuticals at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Aplastic Anemia
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jun 2023to 25 Feb 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Pediatric patients with sAA who initiated eltrombopag and IST treatment in Korea.
Who may be able to join
- 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.
Who cannot join
- None.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Retrospective Chart Review of Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
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.
- Conditions
- Aplastic Anemia
- Keywords
- Severe Aplastic Anemia, Eltrombopag, Retrospective chart review, Safety
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag and IST Treatment Cohort | Pediatric patients with sAA who received first-line eltrombopag in combination with IST. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 6 months
Secondary outcomes (1)
Overall Response Rate (ORR)
ORR includes complete response (CR) and partial response (PR).
CR was defined as meeting all three of the following criteria:
- Absolute neutrophil count (ANC) > 1.0 × 10^9/L
- Platelet count > 100 × 10^9/L
- Hemoglobin > 100 g/L
PR was defined as hematologic improvement that did not meet the criteria for CR, but also did not meet the criteria for sAA.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2026
- Study start21 Jun 2023
- Primary completion25 Feb 2025
- Study completion25 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07415083
- Sponsor's protocol ID
- CETB115GKR02
- Status last verified
- Feb 2026
- First submitted
- 11 Feb 2026
- Last updated
- 19 Feb 2026
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Chart Review of Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07415083
- Acronym / sponsor ID
- CETB115GKR02
- Conditions
- Aplastic Anemia
- Keywords
- Severe Aplastic Anemia, Eltrombopag, Retrospective chart review, Safety
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2023 (actual)
- Primary completion
- 25 Feb 2025 (actual)
- Study completion
- 25 Feb 2025 (actual)
- First posted
- 17 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag and IST Treatment Cohort | – | –Pediatric patients with sAA who received first-line eltrombopag in combination with IST. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Pediatric patients with sAA who initiated eltrombopag and IST treatment in Korea.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- None.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 6 months
Incidence of all AEs/adverse drug reactions (ADRs), SAEs/serious adverse drug reactions (SADRs), and unexpected AEs/ADRs.
1 secondary outcome
Overall Response Rate (ORR)
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2026.
- Link to study resultsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Aplastic Anemia, run by Novartis Pharmaceuticals at 1 site 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 3 inclusion and 1 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 19 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Feb 2026.