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

Last updated: November 27, 2024
Sponsor: Daiichi Sankyo
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

TURALIO™

Clinical Study ID

NCT04635111
PL3397-A-U401
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Adult participants with symptomatic TGCT associated with severe morbidity orfunctional 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 × upperlimit 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 xULN

  • Consent to study procedures, long-term safety follow-up, and use of data from theTURALIO™ (pexidartinib) Risk Evaluation and Mitigation Strategy (REMS) program

Exclusion

Exclusion Criteria:

  • Not applicable

Study Design

Total Participants: 30
Treatment Group(s): 1
Primary Treatment: TURALIO™
Phase:
Study Start date:
January 07, 2021
Estimated Completion Date:
March 01, 2036

Study 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. 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.

Connect with a study center

  • Kamalesh K Sankhala MD INC

    Santa Monica, California 90403
    United States

    Active - Recruiting

  • Sarcoma Oncology Research Center LLC

    Santa Monica, California 90403
    United States

    Site Not Available

  • UCLA Hematology and Oncology

    Santa Monica, California 90404
    United States

    Active - Recruiting

  • The Oncology Institute of Hope and Innovation

    Whittier, California 90602
    United States

    Site Not Available

  • Dana-Farber Cancer Institute

    Boston, Massachusetts 02215
    United States

    Active - Recruiting

  • Montefiore Medical Center

    New York, New York 10467
    United States

    Site Not Available

  • OSU - James Comprehensive Cancer Center

    Columbus, Ohio 43210
    United States

    Active - Recruiting

  • Kelsey Seybold Clinic - Pearland

    Houston, Texas 77014
    United States

    Site Not Available

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