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

NCT04635111 | Recruiting DMC FDA Drug

• 1 other identifier: PL3397-A-U401
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Hepatotoxicity; Tenosynovial Giant Cell Tumor

Keywords

Hepatotoxicity; Tenosynovial Giant Cell Tumor; Pexidartinib; TURALIO™

Outcome Measures

Primary Outcomes (1)

• Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)

  Hepatic failure is defined as severe liver injury with encephalopathy and impaired synthetic function (INR \>= 1.5) as well as liver transplant and hepatic-related death.

  Baseline up to 10 years

Secondary Outcomes (3)

• Number of Participants With Liver Test Abnormalities

  Baseline up to 10 years

• Number of Participants With A Liver Transplant

  Baseline up to 10 years

• Number of Participants Experiencing Death

  Baseline up to 10 years

Study Arms (1)

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).

Drug: TURALIO™

Interventions

TURALIO™ 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.

Symptomatic TGCT Participants

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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 may not qualify if:

• Not applicable

Sponsors & Collaborators

• Daiichi Sankyo: lead

Study Sites (8)

Kamalesh K Sankhala MD INC

Santa Monica, California, 90403, United States

RECRUITING

Sarcoma Oncology Research Center LLC

Santa Monica, California, 90403, United States

NOT YET RECRUITING

UCLA Hematology and Oncology

Santa Monica, California, 90404, United States

RECRUITING

The Oncology Institute of Hope and Innovation

Whittier, California, 90602, United States

WITHDRAWN

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Montefiore Medical Center

New York, New York, 10467, United States

WITHDRAWN

OSU - James Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

RECRUITING

Kelsey Seybold Clinic - Pearland

Houston, Texas, 77014, United States

NOT YET RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

The optional liver biopsy will be performed as soon as reasonably possible after the liver test abnormality criteria are first met and before liver tests have returned to normal. Four blood samples will be drawn on the day of, or up to 4 days before, the optional biopsy. One sample will evaluate liver test results (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], alkaline phosphatase \[ALP\], gammaglutamyl transferase \[GGT\], total bilirubin \[TBIL\], direct bilirubin), serum creatinine, and blood urea nitrogen (BUN) at the study site. The remaining three samples will be used for central laboratory analysis.

MeSH Terms

Conditions

Giant Cell Tumor of Tendon Sheath

Interventions

pexidartinib

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Study Officials

• Global Clinical Leader

  Daiichi Sankyo

  STUDY DIRECTOR

Central Study Contacts

Daiichi Sankyo Contact for Clinical Trial Information

CONTACT

908-992-6400; CTRinfo@dsi.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2020
• First Posted: November 18, 2020
• Study Start: January 7, 2021
• Primary Completion (Estimated): June 1, 2036
• Study Completion (Estimated): June 1, 2036
• Last Updated: October 6, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Studies for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
• Access Criteria: Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.

Locations

US (8)