Een langetermijnstudie ter evaluatie van hepatotoxiciteit die verband houdt met de behandeling met TURALIO™ (Pexidartinib)
Een langetermijnstudie om het risico op hepatotoxiciteit in verband met de behandeling met TURALIO™ (Pexidartinib) verder te evalueren
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.
Studie Overzicht
Toestand
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Contacten en locaties
Studie Locaties
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California
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Santa Monica, California, Verenigde Staten, 90403
- Sarcoma Oncology Research Center LLC
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Santa Monica, California, Verenigde Staten, 90403
- Kamalesh K Sankhala MD INC
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Santa Monica, California, Verenigde Staten, 90404
- UCLA Hematology and Oncology
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Massachusetts
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Boston, Massachusetts, Verenigde Staten, 02215
- Dana-Farber Cancer Institute
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Ohio
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Columbus, Ohio, Verenigde Staten, 43210
- OSU - James Comprehensive Cancer Center
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Texas
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Houston, Texas, Verenigde Staten, 77014
- Kelsey Seybold Clinic - Pearland
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Bemonsteringsmethode
Studie Bevolking
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).
Beschrijving
Inclusion Criteria:
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Adult participants with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery
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Age ≥18 years old
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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
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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
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Observatiemodellen: COHORT
- Tijdsperspectieven: PROSPECTIVE
Cohorten en interventies
Groep / Cohort |
Interventie / Behandeling |
|---|---|
|
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. |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
Tijdsspanne: Baseline up to 10 years
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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
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Aantal deelnemers met een levertransplantatie
Tijdsspanne: Basislijn tot 10 jaar
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Basislijn tot 10 jaar
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Aantal deelnemers dat de dood ervaart
Tijdsspanne: Basislijn tot 10 jaar
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Basislijn tot 10 jaar
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|
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Number of Participants With Liver Test Abnormalities
Tijdsspanne: 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
|
Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Studie directeur: Global Clinical Leader, Daiichi Sankyo
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- PL3397-A-U401
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Beschrijving IPD-plan
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-tijdsbestek voor delen
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-toegangscriteria voor delen
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 delen Ondersteunend informatietype
- LEERPROTOCOOL
- SAP
- MVO
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
product vervaardigd in en geëxporteerd uit de V.S.
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