Revolade Tablets Specified Drug-use Survey

Novartis Pharmaceuticals10 enrolled

Overview

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).

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 Type

OBSERVATIONAL

Enrollment

Conditions

Aplastic Anemia

Interventions

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.

Eligibility

Sex: ALLMin age: 6 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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" Exclusion Criteria: * 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)

Locations (15)

Novartis Investigative Site

Nagoya, Aichi-ken, Japan

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, Japan

Outcomes

Primary Outcomes

Occurrence of serious adverse events

Occurrence of serious adverse events to be provided

Time frame: Up to 1 year

Secondary Outcomes

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

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

Occurrence of clonal evolution \[cytogenetic abnormalities, myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), and paroxysmal nocturnal hemoglobinuria (PNH)\]

Time frame: Up to 1 year

Hematologic response status

Hematologic response status to be provided

Time frame: Up to 1 year

Change over time from baseline in platelet count

Change over time from baseline in platelet count to be provided

Time frame: Baseline, 1 year

Change over time from baseline in hemoglobin

Change over time from baseline in hemoglobin to be provided

Time frame: Baseline, 1 year

Central Contacts

Novartis Pharmaceuticals

CONTACT

+81337978748 novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Data from ClinicalTrials.gov

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