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

NCT ID: NCT04635111

Last Updated: 2025-10-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

30 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-01-07

Study Completion Date

2036-06-01

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hepatotoxicity Tenosynovial Giant Cell Tumor

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Symptomatic TGCT Participants

Adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery, and who experience moderate or severe hepatotoxicity due to use of TURALIO™ (pexidartinib).

TURALIO™

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Daiichi Sankyo

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Leader

Role: STUDY_DIRECTOR

Daiichi Sankyo

Locations

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Kamalesh K Sankhala MD INC

Santa Monica, California, United States

Site Status RECRUITING

Sarcoma Oncology Research Center LLC

Santa Monica, California, United States

Site Status NOT_YET_RECRUITING

UCLA Hematology and Oncology

Santa Monica, California, United States

Site Status RECRUITING

The Oncology Institute of Hope and Innovation

Whittier, California, United States

Site Status WITHDRAWN

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

Montefiore Medical Center

New York, New York, United States

Site Status WITHDRAWN

OSU - James Comprehensive Cancer Center

Columbus, Ohio, United States

Site Status RECRUITING

Kelsey Seybold Clinic - Pearland

Houston, Texas, United States

Site Status NOT_YET_RECRUITING

Countries

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United States

Central Contacts

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Daiichi Sankyo Contact for Clinical Trial Information

Role: CONTACT

[email protected]
908-992-6400

Facility Contacts

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Principal Investigator

Role: primary

Principal Investigator

Role: primary

Principal Investigator

Role: primary

Principal Investigator

Role: primary

Principal Investigator

Role: primary

Principal Investigator

Role: primary

Other Identifiers

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PL3397-A-U401

Identifier Type: -

Identifier Source: org_study_id

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