Evaluating liver damage from TURALIO™ treatment

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

This study is testing the long-term risk of liver damage from TURALIO™ treatment in patients with tenosynovial giant cell tumor who have liver issues.

Quick facts

• Study type: Observational
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Daiichi Sankyo (Industry-sponsored)
• Drugs / interventions: pexidartinib
• Locations: 8 sites (Santa Monica, California and 7 other locations)
• Trial ID: NCT04635111 on ClinicalTrials.gov

What this trial studies

This observational study aims to assess the long-term risk of liver failure associated with TURALIO™ (pexidartinib) in patients with symptomatic tenosynovial giant cell tumor (TGCT) who experience hepatotoxicity. Participants will undergo liver biopsies to analyze immune cell profiles and investigate the mechanisms behind liver injury. Additionally, blood samples will be collected to monitor liver function and conduct pharmacogenomic testing. Patients will be followed for at least 10 years to evaluate the long-term safety and risk of hepatic failure.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with symptomatic TGCT who have experienced liver test abnormalities due to TURALIO™ treatment.

Not a fit: Patients who have not experienced hepatotoxicity from TURALIO™ or those with TGCT amenable to surgical improvement may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide critical insights into the liver safety profile of TURALIO™, potentially improving patient management and treatment outcomes.

How similar studies have performed: While this study is focused on long-term hepatotoxicity evaluation, similar studies assessing drug safety profiles have shown success in identifying risks and improving patient outcomes.

Eligibility criteria

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

Where this trial is running

Santa Monica, California and 7 other locations

• Kamalesh K Sankhala MD INC — Santa Monica, California, United States (Recruiting)
• Sarcoma Oncology Research Center LLC — Santa Monica, California, United States (Not_yet_recruiting)
• UCLA Hematology and Oncology — Santa Monica, California, United States (Recruiting)
• The Oncology Institute of Hope and Innovation — Whittier, California, United States (Withdrawn)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (Recruiting)
• Montefiore Medical Center — New York, New York, United States (Withdrawn)
• OSU - James Comprehensive Cancer Center — Columbus, Ohio, United States (Recruiting)
• Kelsey Seybold Clinic - Pearland — Houston, Texas, United States (Not_yet_recruiting)

Study contacts

• Study coordinator: Daiichi Sankyo Contact for Clinical Trial Information

Conditions Hepatotoxicity; Tenosynovial Giant Cell Tumor; Pexidartinib; TURALIO™