A Long-term Study to Further Evaluate the Risk of Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 8
- 主要终点
- Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •Not applicable
研究组 & 干预措施
Symptomatic TGCT Participants
Adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery, and who experience moderate or severe hepatotoxicity due to use of TURALIO™ (pexidartinib).
干预措施: TURALIO™ (Drug)
结局指标
主要结局
Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
时间窗: Baseline up to 10 years
Hepatic failure is defined as severe liver injury with encephalopathy and impaired synthetic function (INR \>= 1.5) as well as liver transplant and hepatic-related death.
次要结局
- Number of Participants With Liver Test Abnormalities(Baseline up to 10 years)
- Number of Participants With A Liver Transplant(Baseline up to 10 years)
- Number of Participants Experiencing Death(Baseline up to 10 years)