Un estudio a largo plazo que evalúa la hepatotoxicidad asociada con el tratamiento con TURALIO™ (pexidartinib)
Un estudio a largo plazo para evaluar más a fondo el riesgo de hepatotoxicidad asociado con el tratamiento con TURALIO™ (pexidartinib)
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.
Descripción general del estudio
Estado
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Contactos y Ubicaciones
Ubicaciones de estudio
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California
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Santa Monica, California, Estados Unidos, 90403
- Sarcoma Oncology Research Center LLC
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Santa Monica, California, Estados Unidos, 90403
- Kamalesh K Sankhala MD INC
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Santa Monica, California, Estados Unidos, 90404
- UCLA Hematology and Oncology
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02215
- Dana-Farber Cancer Institute
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Ohio
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Columbus, Ohio, Estados Unidos, 43210
- OSU - James Comprehensive Cancer Center
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Texas
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Houston, Texas, Estados Unidos, 77014
- Kelsey Seybold Clinic - Pearland
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-
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
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).
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: PROSPECTIVE
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
|
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. |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Frequency of Hepatic Failure After Discontinuation of TURALIO™ (pexidartinib)
Periodo de tiempo: 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
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Número de participantes con trasplante de hígado
Periodo de tiempo: Línea base hasta 10 años
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Línea base hasta 10 años
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Número de participantes que experimentaron la muerte
Periodo de tiempo: Línea base hasta 10 años
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Línea base hasta 10 años
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|
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Number of Participants With Liver Test Abnormalities
Periodo de tiempo: 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
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Global Clinical Leader, Daiichi Sankyo
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades musculoesqueléticas
- Enfermedades Musculares
- Neoplasias
- Enfermedades Articulares
- Neoplasias por tipo histológico
- Neoplasias De Tejidos Conectivos Y Blandos
- Neoplasias Del Tejido Conectivo
- Sinovitis
- Tendinopatía
- Tumores de células gigantes
- Tumor de células gigantes de la vaina del tendón
- pexidartinib
Otros números de identificación del estudio
- PL3397-A-U401
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
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/
Marco de tiempo para compartir IPD
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.
Criterios de acceso compartido de IPD
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.
Tipo de información de apoyo para compartir IPD
- PROTOCOLO DE ESTUDIO
- SAVIA
- RSC
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
producto fabricado y exportado desde los EE. UU.
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