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.
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.
Study Type
OBSERVATIONAL
Enrollment
30
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.
Kamalesh K Sankhala MD INC
Santa Monica, California, United States
RECRUITINGSarcoma Oncology Research Center LLC
Santa Monica, California, United States
NOT_YET_RECRUITINGUCLA Hematology and Oncology
Santa Monica, California, United States
Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
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.
Time frame: Baseline up to 10 years
Number of Participants With Liver Test Abnormalities
Liver test abnormalities were defined as the following: 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.
Time frame: Baseline up to 10 years
Number of Participants With A Liver Transplant
Time frame: Baseline up to 10 years
Number of Participants Experiencing Death
Time frame: Baseline up to 10 years
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The Oncology Institute of Hope and Innovation
Whittier, California, United States
WITHDRAWNDana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGMontefiore Medical Center
New York, New York, United States
WITHDRAWNOSU - James Comprehensive Cancer Center
Columbus, Ohio, United States
RECRUITINGKelsey Seybold Clinic - Pearland
Houston, Texas, United States
NOT_YET_RECRUITING