Revolade Tablets Specified Drug-use Survey

Официальное название

Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)

Обзор

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).

Подробное описание

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.

Вмешательства

• Другое eltrombopag
  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.

Первичные конечные точки

• Occurrence of serious adverse events [Срок оценки: Up to 1 year]

Вторичные конечные точки (10)

• Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation [Срок оценки: Up to 1 Year]
• Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies) [Срок оценки: Up to 1 year]
• Occurrence of clonal evolution [Срок оценки: Up to 1 year]
• Hematologic response status [Срок оценки: Up to 1 year]
• Change over time from baseline in platelet count [Срок оценки: Baseline, 1 year]
• Change over time from baseline in hemoglobin [Срок оценки: Baseline, 1 year]
• Change over time from baseline in neutrophil count [Срок оценки: Baseline, 1 year]
• Change over time from baseline in reticulocyte count [Срок оценки: Baseline, 1 year]
• Change over time from baseline in transfusion dependence [Срок оценки: Baseline, 1 year]
• Change over time from baseline in transfusion volume [Срок оценки: Baseline, 1 year]

Критерии участия

Критерии включения

• 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"

Критерии исключения

• 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)

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Япония · 17 центров

• Novartis Investigative Site — Nagoya
• Novartis Investigative Site — Nagoya
• Novartis Investigative Site — Toyoake
• Novartis Investigative Site — Chiba
• Novartis Investigative Site — Amagasaki
• Novartis Investigative Site — Kobe
• Novartis Investigative Site — Matsumoto
• Novartis Investigative Site — Shimajiri-Gun
• … и ещё 9 центров

Идентификаторы

NCT: NCT06287268 · CETB115G1401

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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