Aplastic AnemiaNCT06287268
Revolade Tablets Specified Drug-use Survey
An observational study of Aplastic Anemia, run by Novartis Pharmaceuticals at 17 sites in Japan.
Trial summary
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA). Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- eltrombopag
- How many people
- 10 participants
- Where
- 17 sitesJapan
- Study dates
- 17 Jul 2024to 31 Oct 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: The survey will be conducted in pediatric patients with Aplastic anemia (AA) who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA."
Who may be able to join
- Patients whose legally acceptable representative has given written consent for cooperation in this survey prior to enrollment in this survey
- Patients aged ≥ 6 years and < 18 years at the start of treatment with eltrombopag
- Pediatric patients with AA who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA"
Who cannot join
- Patients who have received ATG without concomitant use of eltrombopag
- Patients with congenital AA
- Patients with suspected or confirmed diagnosis of myelodysplastic syndrome (MDS) at the start of treatment with eltrombopag
- Patients who have received any drug products containing the same ingredient as eltrombopag (including investigational products)
Trial locations and contacts
17 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
17 sites in 1 country
Japan17 sites, 17 recruiting
- Recruiting
Novartis Investigative Site
Amagasaki, Hyōgo, 660 8550
- Recruiting
Novartis Investigative Site
Chiba, Chiba, 260 8677
- Recruiting
Novartis Investigative Site
Chuo Ku, Tokyo, 1048560
- Recruiting
Novartis Investigative Site
Izumi, Osaka, 5941101
- Recruiting
Novartis Investigative Site
Kobe, Hyōgo, 6500047
- Recruiting
Novartis Investigative Site
Matsumoto, Nagano, 3908621
- Recruiting
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511
- Recruiting
Novartis Investigative Site
Nagoya, Aichi-ken, 4668560
- Recruiting
Novartis Investigative Site
Ohtsu, Shiga, 5202192
- Recruiting
Novartis Investigative Site
Osaka, Osaka, 5340021
- Recruiting
Novartis Investigative Site
Ōta-ku, Tokyo, 143 8541
- Recruiting
Novartis Investigative Site
Saitama, Saitama, 3308777
- Recruiting
Novartis Investigative Site
Sakai, Osaka, 590-0197
- Recruiting
Novartis Investigative Site
Setagaya-ku, Tokyo, 1578535
- Recruiting
Novartis Investigative Site
Shimajiri-Gun, Okinawa, 901-1303
- Recruiting
Novartis Investigative Site
Toyoake, Aichi-ken, 4701192
- Recruiting
Novartis Investigative Site
Wakayama, Wakayama, 641-8510
About this study
Official title: Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
Read the detailed description
The objective of this survey is to confirm the safety and efficacy of eltrombopag in ATG treatment naive pediatric patients with AA. Eltrombopag should be administered according to the dosage and administration specified in the latest version of the package insert. The observation period is 1 year (364 days) from the start of treatment with this product, regardless of whether treatment with eltrombopag is continued or not. However, if hematopoietic stem cell transplantation is performed within 1 year after the start of treatment with eltrombopag, the observation period shall be until the date of hematopoietic stem cell transplantation.
- Conditions
- Aplastic Anemia
- Being tested
- eltrombopag
- Keywords
- NIS, Pediatric Aplastic Anemia, AA, Revolade Tablets, eltrombopag, Drug-use Survey
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eltrombopagOther
This is an observational study. There is no treatment allocation. After confirming that patients are fulfilling the eligibility criteria, patients will be registered in this survey.
| Study group | Role | What they receive |
|---|---|---|
| eltrombopag | ATG treatment naïve pediatric patients with AA in routine clinical practice Receives: Other: eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of serious adverse events
Occurrence of serious adverse events to be provided
Measured: Up to 1 year
Secondary outcomes (10)
Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation
Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation to be provided
Measured: Up to 1 Year
Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)
Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)
Measured: Up to 1 year
Occurrence of clonal evolution
Occurrence of clonal evolution [cytogenetic abnormalities, myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), and paroxysmal nocturnal hemoglobinuria (PNH)]
Measured: Up to 1 year
Hematologic response status
Hematologic response status to be provided
Measured: Up to 1 year
Change over time from baseline in platelet count
Change over time from baseline in platelet count to be provided
Measured: Baseline, 1 year
Change over time from baseline in hemoglobin
Change over time from baseline in hemoglobin to be provided
Measured: Baseline, 1 year
Change over time from baseline in neutrophil count
Change over time from baseline in neutrophil count to be provided
Measured: Baseline, 1 year
Change over time from baseline in reticulocyte count
Change over time from baseline in reticulocyte count to be provided
Measured: Baseline, 1 year
Change over time from baseline in transfusion dependence
Change over time from baseline in transfusion dependence to be provided
Measured: Baseline, 1 year
Change over time from baseline in transfusion volume
Change over time from baseline in transfusion volume to be provided
Measured: Baseline, 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2024
- Study start17 Jul 2024
- Primary completion31 Oct 2028 (estimated)
- Study completion31 Oct 2028 (estimated)
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
- Other
- Enrolment
- 10 participants (estimated)
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
- NCT06287268
- Sponsor's protocol ID
- CETB115G1401
- Status last verified
- Feb 2026
- First submitted
- 22 Feb 2024
- 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
- Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)
- ClinicalTrials.gov ID
- NCT06287268
- Acronym / sponsor ID
- CETB115G1401
- Conditions
- Aplastic Anemia
- Keywords
- NIS, Pediatric Aplastic Anemia, AA, Revolade Tablets, eltrombopag, Drug-use Survey
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Jul 2024 (actual)
- Primary completion
- 31 Oct 2028 (estimated)
- Study completion
- 31 Oct 2028 (estimated)
- First posted
- 1 Mar 2024 (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.
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
Detailed description
The objective of this survey is to confirm the safety and efficacy of eltrombopag in ATG treatment naive pediatric patients with AA. Eltrombopag should be administered according to the dosage and administration specified in the latest version of the package insert. The observation period is 1 year (364 days) from the start of treatment with this product, regardless of whether treatment with eltrombopag is continued or not. However, if hematopoietic stem cell transplantation is performed within 1 year after the start of treatment with eltrombopag, the observation period shall be until the date of hematopoietic stem cell transplantation.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| eltrombopag | – | Other: eltrombopagATG treatment naïve pediatric patients with AA in routine clinical practice |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The survey will be conducted in pediatric patients with Aplastic anemia (AA) who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA."
Key inclusion criteria (3 listed)
- Patients whose legally acceptable representative has given written consent for cooperation in this survey prior to enrollment in this survey
- Patients aged ≥ 6 years and < 18 years at the start of treatment with eltrombopag
- Pediatric patients with AA who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA"
Key exclusion criteria (4 listed)
- Patients who have received ATG without concomitant use of eltrombopag
- Patients with congenital AA
- Patients with suspected or confirmed diagnosis of myelodysplastic syndrome (MDS) at the start of treatment with eltrombopag
- Patients who have received any drug products containing the same ingredient as eltrombopag (including investigational products)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| eltrombopagThis is an observational study. There is no treatment allocation. After confirming that patients are fulfilling the eligibility criteria, patients will be registered in this survey. | Other | eltrombopag |
Primary outcome
Occurrence of serious adverse events
Time frame: Up to 1 year
Occurrence of serious adverse events to be provided
10 secondary outcomes
Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation
Time frame: Up to 1 Year
Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)
Time frame: Up to 1 year
Occurrence of clonal evolution
Time frame: Up to 1 year
Hematologic response status
Time frame: Up to 1 year
Change over time from baseline in platelet count
Time frame: Baseline, 1 year
Change over time from baseline in hemoglobin
Time frame: Baseline, 1 year
Change over time from baseline in neutrophil count
Time frame: Baseline, 1 year
Change over time from baseline in reticulocyte count
Time frame: Baseline, 1 year
Change over time from baseline in transfusion dependence
Time frame: Baseline, 1 year
Change over time from baseline in transfusion volume
Time frame: Baseline, 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2026.
- 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.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
17 sites 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 17 sites in Japan.
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 4 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: looking for participants now, 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 17 sites in Japan. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+81337978748, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of serious adverse events (time frame: Up to 1 year). 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.