Revolade Tablets Specified Drug-use Survey
- Conditions
- Aplastic Anemia
- Interventions
- Other: eltrombopag
- Registration Number
- NCT06287268
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief 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).
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 10
- 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"
- 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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description eltrombopag eltrombopag ATG treatment naïve pediatric patients with AA in routine clinical practice
- Primary Outcome Measures
Name Time Method Occurrence of serious adverse events Up to 1 year Occurrence of serious adverse events to be provided
- Secondary Outcome Measures
Name Time Method Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation Up to 1 Year Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation to be provided
Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies) Up to 1 year Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies)
Change over time from baseline in hemoglobin Baseline, 1 year Change over time from baseline in hemoglobin to be provided
Change over time from baseline in transfusion volume Baseline, 1 year Change over time from baseline in transfusion volume to be provided
Occurrence of clonal evolution Up to 1 year Occurrence of clonal evolution \[cytogenetic abnormalities, myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), and paroxysmal nocturnal hemoglobinuria (PNH)\]
Hematologic response status Up to 1 year Hematologic response status to be provided
Change over time from baseline in platelet count Baseline, 1 year Change over time from baseline in platelet count to be provided
Change over time from baseline in neutrophil count Baseline, 1 year Change over time from baseline in neutrophil count to be provided
Change over time from baseline in reticulocyte count Baseline, 1 year Change over time from baseline in reticulocyte count to be provided
Change over time from baseline in transfusion dependence Baseline, 1 year Change over time from baseline in transfusion dependence to be provided
Trial Locations
- Locations (1)
Novartis Investigative Site
🇯🇵Saitama, Japan