Идёт набор NCT04635111

A Long-term Study Evaluating Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment

— Наблюдательное Hepatotoxicity Tenosynovial Giant Cell Tumor

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: TURALIO™.
Кому может быть актуально: Состояния в реестре: Hepatotoxicity, Tenosynovial Giant Cell Tumor. Базовые параметры: от 18 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Long-term Study to Further Evaluate the Risk of Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment

Обзор

A study to evaluate the long-term risk of hepatic failure with TURALIO™ (pexidartinib) and the mechanism of liver injury based upon optional liver biopsy information among patients who received or are receiving TURALIO™ (pexidartinib) and experience hepatotoxicity.

Подробное описание

This FDA post-marketing requirement study will evaluate the long-term risk of hepatic failure with TURALIO™ (pexidartinib) and the mechanism of liver injury based upon liver biopsy information among patients who received or are receiving TURALIO™ (pexidartinib) for symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery, and who experience hepatotoxicity. An optional liver biopsy will be collected from enrolled patients for central laboratory analysis of macrophage and immune cell profiles to investigate possible mechanisms of the hepatotoxicity. Additionally, 4 blood samples will be collected to evaluate liver function, other relevant safety tests, peripheral immune cells, and for pharmacogenomic testing. Enrolled patients will be followed at least yearly for 10 years to assess long-term risk of hepatic failure.

Вмешательства

Препарат TURALIO™
This is a non-interventional, observational study to evaluate long-term risk of hepatic failure associated with TURALIO™ (pexidartinib) treatment. No study medication will be provided to the participants.

Первичные конечные точки

Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib) [Срок оценки: Baseline up to 10 years]

Вторичные конечные точки (3)

Number of Participants With Liver Test Abnormalities [Срок оценки: Baseline up to 10 years]
Number of Participants With A Liver Transplant [Срок оценки: Baseline up to 10 years]
Number of Participants Experiencing Death [Срок оценки: Baseline up to 10 years]

Критерии участия

Критерии включения

Adult participants with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery
Age ≥18 years old
Emergence of at least one of the following liver test abnormalities due to TURALIO™ (pexidartinib) exposure:

Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) with concurrent total bilirubin (TBIL) >2 × ULN
Isolated TBIL >2 × ULN (excluding patients with Gilbert's syndrome)
Isolated AST or ALT >10 × ULN
Alkaline phosphatase (ALP) >2 x ULN with gamma-glutamyl transferase (GGT) >2 x ULN
Consent to study procedures, long-term safety follow-up, and use of data from the TURALIO™ (pexidartinib) Risk Evaluation and Mitigation Strategy (REMS) program