- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04635111
A Long-term Study Evaluating Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment
September 13, 2023 updated by: Daiichi Sankyo, Inc.
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 liver biopsy information among patients who received or are receiving TURALIO™ (pexidartinib) and experience hepatotoxicity.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
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.
Study Type
Observational
Enrollment (Estimated)
30
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Daiichi Sankyo Contact for Clinical Trial Information
- Phone Number: 908-992-6400
- Email: CTRinfo@dsi.com
Study Locations
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California
-
Santa Monica, California, United States, 90404
- Recruiting
- UCLA Hematology and Oncology
-
Contact:
- Principal Investigator
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Santa Monica, California, United States, 90403
- Recruiting
- Kamalesh K Sankhala MD INC
-
Contact:
- Principal Investigator
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Whittier, California, United States, 90602-3171
- Withdrawn
- The Oncology Institute of Hope and Innovation
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Recruiting
- Dana-Farber Cancer Institute
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Contact:
- Principal Investigator
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New York
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New York, New York, United States, 10467
- Withdrawn
- Montefiore Medical Center
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Ohio
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Columbus, Ohio, United States, 43210
- Recruiting
- OSU - James Comprehensive Cancer Center
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Contact:
- Principal Investigator
-
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
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).
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
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).
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
Baseline up to 10 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Liver Test Abnormalities
Time Frame: Baseline up to 10 years
|
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.
|
Baseline up to 10 years
|
Number of Participants With A Liver Transplant
Time Frame: Baseline up to 10 years
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Baseline up to 10 years
|
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Number of Participants Experiencing Death
Time Frame: Baseline up to 10 years
|
Baseline up to 10 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Global Clinical Leader, Daiichi Sankyo, Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 7, 2021
Primary Completion (Estimated)
June 1, 2025
Study Completion (Estimated)
March 1, 2036
Study Registration Dates
First Submitted
October 29, 2020
First Submitted That Met QC Criteria
November 17, 2020
First Posted (Actual)
November 18, 2020
Study Record Updates
Last Update Posted (Actual)
September 14, 2023
Last Update Submitted That Met QC Criteria
September 13, 2023
Last Verified
September 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PL3397-A-U401
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD Plan Description
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/
IPD Sharing 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.
IPD Sharing 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.
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Tenosynovial Giant Cell Tumor
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Deciphera Pharmaceuticals LLCActive, not recruitingTenosynovial Giant Cell Tumor | Giant Cell Tumor of Tendon Sheath | Pigmented Villonodular Synovitis | Tenosynovial Giant Cell Tumor, Diffuse | Tenosynovial Giant Cell Tumor, LocalizedNorway, United States, France, Spain, Hong Kong, Netherlands, Italy, Germany, Australia, United Kingdom, Canada, Poland, Switzerland
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Deciphera Pharmaceuticals LLCActive, not recruitingAdvanced Malignant Neoplasm | Tenosynovial Giant Cell Tumor | Giant Cell Tumor of Tendon Sheath | Pigmented Villonodular Synovitis | Tenosynovial Giant Cell Tumor, DiffuseUnited States, France, Spain, United Kingdom, Netherlands, Canada, Italy, Australia, Poland
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Novartis PharmaceuticalsCompletedPigmented Villonodular Synovitis | PVNS | Giant Cell Tumor of the Tendon Sheath | GCCTS | Tenosynovial Giant Cell Tumor Localized or Diffused Type | GCTSUnited States, Switzerland
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Daiichi Sankyo Co., Ltd.Active, not recruitingTenosynovial Giant Cell TumorTaiwan, China
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Andrew J. Wagner, MD, PhDMassachusetts General Hospital; Novartis; Brigham and Women's HospitalActive, not recruitingTenosynovial Giant Cell Tumor | Pigmented Villonodular Synovitis | Diffuse-type Giant Cell TumorUnited States
-
Daiichi Sankyo, Inc.CompletedTenosynovial Giant Cell TumorUnited States, Spain, Taiwan, Australia, Hungary, Italy, Netherlands
-
Daiichi Sankyo Co., Ltd.RecruitingTenosynovial Giant Cell TumorJapan
-
Abbisko Therapeutics Co, LtdRecruitingNeoplasms | Tenosynovial Giant Cell TumorUnited States
-
SynOx Therapeutics LimitedActive, not recruiting
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Daiichi Sankyo, Inc.OptimapharmCompletedGiant Cell TumorsUnited States, France, Spain, Netherlands, Italy, Austria, United Kingdom, Germany
Clinical Trials on TURALIO™
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Daiichi Sankyo Co., Ltd.Active, not recruitingTenosynovial Giant Cell TumorTaiwan, China
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Daiichi Sankyo Co., Ltd.RecruitingTenosynovial Giant Cell TumorJapan
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National Cancer Institute (NCI)RecruitingSarcoma | Precursor Cell Lymphoblastic Leukemia-Lymphoma | Neurofibroma, Plexiform | Leukemia, Promyelocytic, AcuteUnited States
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Daiichi Sankyo, Inc.CompletedTenosynovial Giant Cell TumorUnited States, Spain, Taiwan, Australia, Hungary, Italy, Netherlands
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BaroNova, Inc.Completed
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London Health Sciences CentreUniversity of Western Ontario, Canada; Synaptive MedicalUnknownBrain Damage, Chronic | Cerebellar Cognitive Affective Syndrome | Cerebellar Mutism
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Asklepios Kliniken Hamburg GmbHUniversity of KielCompletedAirway Management | Laryngeal Mask Airway | Fibreoptic Intubation
-
CereVasc IncAlvaMed, Inc.; Simplified Clinical Data Systems, LLC; Bioscience Consulting,...RecruitingHydrocephalus | Hydrocephalus, CommunicatingArgentina
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Medtronic CardiovascularActive, not recruitingAortic AneurysmGermany, New Zealand, United States, Netherlands, Switzerland, United Kingdom, Spain, Australia, Italy, Sweden, France, Austria, Slovakia
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Gülçin Özalp GerçekerCompletedCatheter Complications | Catheter LeakageTurkey