Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

Summary

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.

Official Title

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

Details

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. A 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.

Keywords

Hepatotoxicity Tenosynovial Giant Cell Tumor Pexidartinib TURALIO™ Giant Cell Tumors Giant Cell Tumor of Tendon Sheath

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • Not applicable

Locations

  • UCLA Hematology and Oncology accepting new patients
    Santa Monica California 90404 United States
  • The Oncology Institute of Hope and Innovation accepting new patients
    Whittier California 90602-3171 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Daiichi Sankyo, Inc.
ID
NCT04635111
Study Type
Observational [Patient Registry]
Participants
Expecting 30 study participants
Last Updated