Clinical Trials - DataSource by Daiichi Sankyo

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

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.

CONDITIONS

Hepatotoxicity, Tenosynovial Giant Cell Tumor

PRODUCT

Pexidartinib

GENDER

ALL

AGE

18 Years

SPONSOR

Daiichi Sankyo

COLLABORATOR

TRIAL RUNS IN

8 Global Locations

Trial Summary

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.

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

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

Not applicable

This was last updated on 2025-11-06 19:34:37.034.

The content contained is taken is taken directly from public registry ClinicalTrials.gov and has not been edited.

Information about pipeline compounds or investigational uses of compounds does not imply FDA approval for these compounds or uses, nor does it establish the safety or efficacy of these compounds or uses. There is no guarantee that the pipeline compounds or investigational uses will receive FDA approval. Daiichi Sankyo does not recommend or suggest use of its medicines in a manner inconsistent with FDA-approved labeling.