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Recruiting NCT04635111

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

— Observational Hepatotoxicity Tenosynovial Giant Cell Tumor

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: TURALIO™.
Who it may be relevant to
Registry conditions: Hepatotoxicity, Tenosynovial Giant Cell Tumor. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Detailed description

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.

Interventions

  • Drug TURALIO™
    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.

Primary outcome measures

  • Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib) [Time frame: Baseline up to 10 years]
Secondary outcome measures (3)
  • Number of Participants With Liver Test Abnormalities [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]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 8 centers
  • Kamalesh K Sankhala MD INC — Santa Monica
  • Sarcoma Oncology Research Center LLC — Santa Monica
  • UCLA Hematology and Oncology — Santa Monica
  • The Oncology Institute of Hope and Innovation — Whittier
  • Dana-Farber Cancer Institute — Boston
  • Montefiore Medical Center — New York
  • OSU - James Comprehensive Cancer Center — Columbus
  • Kelsey Seybold Clinic - Pearland — Houston

Identifiers

NCT: NCT04635111 · PL3397-A-U401

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗