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

ConditionsHepatotoxicityTenosynovial Giant Cell Tumor
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorDaiichi Sankyo

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult participants with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery

Age ≥18 years old

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)

Disqualifiers

Not applicable

Trial 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).

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • 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.

Treatment groups

30 Participants
are divided into 1 treatment group
Group A: Symptomatic TGCT Participants1 intervention

Trial outcomes

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.

Time frame
Baseline up to 10 years

Secondary outcomes

1

Number of Participants With Liver Test Abnormalities

Liver test abnormalities were defined as the following: 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.

Time frame
Baseline up to 10 years
2

Number of Participants With A Liver Transplant

Time frame
Baseline up to 10 years
3

Number of Participants Experiencing Death

Time frame
Baseline up to 10 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Daiichi Sankyo

Lead sponsor

Source ClinicalTrials.gov
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