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

PL3397-A-U401

A study to 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) and experience hepatotoxicity.

2021-01-07

2025-06-01

2036-03-01

Recruiting

30

Inclusion Criteria: * 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 Exclusion Criteria: * Not applicable

{'studyType': 'OBSERVATIONAL', 'patientRegistry': True, 'targetDuration': '10 Years', 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'The liver biopsy will be performed as soon as reasonably possible after the liver test abnormality criteria are first met and before liver tests have returned to normal. Four blood samples will be drawn on the day of, or up to 4 days before, the biopsy. One sample will evaluate liver test results (alanine aminotransferase \\[ALT\\], aspartate aminotransferase \\[AST\\], alkaline phosphatase \\[ALP\\], gammaglutamyl transferase \\[GGT\\], total bilirubin \\[TBIL\\], direct bilirubin), serum creatinine, and blood urea nitrogen (BUN) at the study site. The remaining three samples will be used for central laboratory analysis.'}, 'enrollmentInfo': {'count': 30, 'type': 'ESTIMATED'}}

2023-09-14