Frequency and Clinical Phenotype of BAP1 Hereditary Predisposition Syndrome
Uveal MelanomaCutaneous Melanoma6 moreThis research will have a significant impact on the overall management of those cancer patients and their family members who are at risk for hereditary cancer due to germline inactivation of BAP1. Our study will ultimately facilitate the development of novel screening, prevention and treatment strategies for these individuals with the syndrome. Because the vast majority of UM develop in pre-existing nevi, characterization of individuals at high risk for development of UM will allow closer screening and earlier intervention which would improve the treatment outcome not only for retaining vision but also for overall survival. Similarly in patients with germline BAP1 mutation CM develops in premalignant atypical melanocytic lesions and careful follow up of these patients will improve the outcome of their disease. In addition this study could have impact on the management of patients with personal and/or family history of several other cancers reported in patients with germline BAP1 mutation such as mesothelioma, renal cell carcinoma, cholangiocarcinoma, hepatocellular carcinoma, meningioma and basal cell carcinoma.
MRI-based Clinico-radiomics Predicting Lymph Node Metastasis Status of Hilar Cholangiocarcinoma...
Hilar CholangiocarcinomaThis is a prospective, observational diagnostic study aiming to assess multiparametric MRI-based clinico-radiomics for identifying lymph node metastasis status in hilar cholangiocarcinoma.
Liver and Biliary Tumor Tissue Registry
Hepatocellular CarcinomaCholangiocarcinomaPatients having surgery to remove a liver or biliary tissue mass or having a biopsy of a mass or lesion will have a tissue sample collected and stored for future research of liver and biliary diseases. A blood sample may also be collected at the time of enrollment.
Melphalan for Use With the Hepatic Delivery System Treatment in Patients With Unresectable Hepatocellular...
Hepatocellular CarcinomaIntrahepatic CholangiocarcinomaThis is a two arm, open label, multi-center, Phase 2 study to evaluate the efficacy and safety of Melphalan/HDS in patients with unresectable Hepatocellular Carcinoma (HCC) or Intra Hepatic Cholangiocarcinoma (ICC) confined to the liver.
An Open-Label Safety and Tolerability Study of INCB062079 in Subjects With Advanced Hepatocellular...
Hepatocellular Carcinoma (HCC)Cholangiocarcinoma4 moreThe purpose of this study is to evaluate the safety and tolerability, and determine the maximum tolerated dose of INCB062079 in subjects with advanced hepatocellular carcinoma and other malignancies.
Phase I/IIa Study to Evaluate the Safety, PK, PD, and Preliminary Efficacy of PLX8394 in Patients...
MelanomaThyroid Cancer5 moreThe study objective is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered PLX8394 in patients with advanced solid tumors. An additional objective is to identify a Recommended Phase 2 (RP2D) for further evaluation in the Extension Cohorts (Phase IIa portion). The study objective of the Extension Cohorts (PART 2 portion) is to assess the objective tumor response and the PK, PD, and safety of PLX8394 when the RP2D is used in patients with advanced BRAF-mutated cancers.
A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma
Advanced CholangiocarcinomaFGFR2 Gene MutationThis is a multi-center, open label, single arm phase II study evaluating BGJ398 (infigratinib) anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor (FGFR) genetic alterations.
Second-line Therapy of Unresectable Cholangiocarcinoma by RADIOEMBOLIZATION
CholestasisProgressive Familial Intrahepatic 3Cholangiocarcinoma (CCK) is a rare tumor (2000 new cases/year in France) with very poor prognosis (overall survival < 3% at 5 years). Less than 20% of patients may benefit from curative surgical resection and most patients have medical treatment by palliative treatment by palliative chemotherapy. It is not standard first-line chemotherapy validated for unresectable CCK, but the best objective response rate (OR) and overall survival (OS) are observed with gemcitabine and platinum associations (OR 24 to 36% and OS between 9.5 to 15.4 months). In case of tumor progression ater this first line therapy, no treatment is currently being validated. RADIOEMBOLIZATION (RE) is a new, transarterial approach to radiation therapy using 90 Yttrium microspheres. In the patients with unresectable CCK , the first pilot studies showed interesting results with rates of OR 45 to 90% and a median OS of 14.9 mots and an acceptable safety. Study Hypothesis : RE could help achieve tumor stabilization in patients with intra-hepatic CCK in tumor progression after first-line therapy.
A Study of Combination of Gemcitabine, Oxaliplatin (GEMOX)-Sorafenib in Patients With Advanced Biliary...
CholangiocarcinomaBiliary Tract Cancer1 moreThe purpose of this study is to build on the efficacy of the GEMOX regimen by adding Sorafenib in the treatment of Biliary Tract Cancer. Since there is no data on the combination of these three agents, the investigators plan to evaluate the safety in a run-in phase I portion in order to define the recommended phase II dose (RPTD). The phase II trial will enroll 40 patients at the RPTD level within 2 years in order to provide a preliminary estimate of progression-free survival (primary endpoint of the trial) in the target population.
Bortezomib in Treating Patients With Unresectable Locally Advanced or Metastatic Adenocarcinoma...
Adenocarcinoma of the Extrahepatic Bile DuctAdenocarcinoma of the Gallbladder8 moreThis phase II trial is studying how well bortezomib works as first-line systemic therapy in treating patients with unresectable locally advanced or metastatic adenocarcinoma (cancer) of the bile duct or gallbladder. Bortezomib may stop the growth of tumor cells by blocking the enzymes necessary for their growth.