A Long-term Study Evaluating Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
Purpose
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.
Conditions
- Hepatotoxicity
- Tenosynovial Giant Cell Tumor
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Study Type
- Observational [Patient Registry]
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| 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). |
|
Recruiting Locations
Santa Monica 5393212, California 5332921 90403
Principal Investigator
Santa Monica 5393212, California 5332921 90404
Principal Investigator
Boston 4930956, Massachusetts 6254926 02215
Principal Investigator
Columbus 4509177, Ohio 5165418 43210
Principal Investigator
More Details
- NCT ID
- NCT04635111
- Status
- Recruiting
- Sponsor
- Daiichi Sankyo
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. 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.