HepatotoxicityNCT04635111
A Long-term Study Evaluating Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
An observational study of Hepatotoxicity and Tenosynovial Giant Cell Tumor, run by Daiichi Sankyo at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatotoxicity, Tenosynovial Giant Cell Tumor
- What is being tested?
- TURALIO™
- How many people
- 30 participants
- Where
- 8 sitesUnited States
- Study dates
- 7 Jan 2021to 1 Jun 2036
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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).
Who may be able to join
- 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
Who cannot join
- Not applicable
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 6 recruiting
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Principal Investigator
- Recruiting
OSU - James Comprehensive Cancer Center
Columbus, Ohio, 43210
Principal Investigator
- Not yet recruiting
Kelsey Seybold Clinic - Pearland
Houston, Texas, 77014
Principal Investigator
- Withdrawn
Montefiore Medical Center
New York, New York, 10467
- Recruiting
Kamalesh K Sankhala MD INC
Santa Monica, California, 90403
Principal Investigator
- Not yet recruiting
Sarcoma Oncology Research Center LLC
Santa Monica, California, 90403
Principal Investigator
- Recruiting
UCLA Hematology and Oncology
Santa Monica, California, 90404
Principal Investigator
- Withdrawn
The Oncology Institute of Hope and Innovation
Whittier, California, 90602
About this study
Official title: A Long-term Study to Further Evaluate the Risk of Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
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.
Read the 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. 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
- Hepatotoxicity, Tenosynovial Giant Cell Tumor
- Being tested
- TURALIO™
- Keywords
- Hepatotoxicity, Tenosynovial Giant Cell Tumor, Pexidartinib, TURALIO™
- Medical subject headings
- Giant Cell Tumor of Tendon Sheath
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: TURALIO™ |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline up to 10 years
Secondary outcomes (3)
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.
Measured: Baseline up to 10 years
Number of Participants With A Liver Transplant
Measured: Baseline up to 10 years
Number of Participants Experiencing Death
Measured: Baseline up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2020
- Study start7 Jan 2021
- Primary completion1 Jun 2036 (estimated)
- Study completion1 Jun 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HepatotoxicityStudies of the same condition, open and under way.
- More trials run by Daiichi SankyoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Daiichi Sankyo
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Leader, Study director, Daiichi Sankyo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04635111
- Sponsor's protocol ID
- PL3397-A-U401
- Status last verified
- Sep 2025
- First submitted
- 29 Oct 2020
- Last updated
- 6 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Study to Further Evaluate the Risk of Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
- ClinicalTrials.gov ID
- NCT04635111
- Acronym / sponsor ID
- PL3397-A-U401
- Conditions
- Hepatotoxicity, Tenosynovial Giant Cell Tumor
- Keywords
- Hepatotoxicity, Tenosynovial Giant Cell Tumor, Pexidartinib, TURALIO™
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Daiichi SankyoIndustry (pharma, biotech, devices)
- Study start
- 7 Jan 2021 (actual)
- Primary completion
- 1 Jun 2036 (estimated)
- Study completion
- 1 Jun 2036 (estimated)
- First posted
- 18 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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. 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Symptomatic TGCT Participants | – | Drug: TURALIO™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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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).
Key inclusion criteria (8 listed)
- 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:
- Consent to study procedures, long-term safety follow-up, and use of data from the TURALIO™ (pexidartinib) Risk Evaluation and Mitigation Strategy (REMS) program
Key exclusion criteria (1 listed)
- Not applicable
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | Symptomatic TGCT Participants |
Primary outcome
Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
Time frame: Baseline up to 10 years
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.
3 secondary outcomes
Number of Participants With Liver Test Abnormalities
Time frame: Baseline up to 10 years
Number of Participants With A Liver Transplant
Time frame: Baseline up to 10 years
Number of Participants Experiencing Death
Time frame: Baseline up to 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2025.
- Individual participant dataSharing planned: Yes. De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Clinical Leader study director
Daiichi Sankyo
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hepatotoxicity and Tenosynovial Giant Cell Tumor, run by Daiichi Sankyo at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Daiichi Sankyo Contact for Clinical Trial Information (908-992-6400, CTRinfo@dsi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib) (time frame: Baseline up to 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2025.