Futibatinib and Pembrolizumab for the Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer
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|ClinicalTrials.gov Identifier: NCT04828486|
Recruitment Status : Recruiting
First Posted : April 2, 2021
Last Update Posted : November 10, 2021
|Condition or disease||Intervention/treatment||Phase|
|Advanced Hepatocellular Carcinoma BCLC Stage A Hepatocellular Carcinoma BCLC Stage B Hepatocellular Carcinoma BCLC Stage C Hepatocellular Carcinoma Metastatic Hepatocellular Carcinoma||Drug: Futibatinib Biological: Pembrolizumab Other: Quality-of-Life Assessment||Phase 2|
I. Determine the efficacy of combination of futibatinib and pembrolizumab in patients with advanced hepatocellular carcinoma (HCC) and high FGF19 expression who has received at least one line of therapy using progression free survival (PFS) at 6 months.
I. Assess the safety and tolerability of futibatinib and pembrolizumab combination through adverse event monitoring.
II. Determine the overall objective response rate (ORR) and overall survival (OS) of patients with advanced HCC treated with futibatinib and pembrolizumab combination.
III. Assess change in overall health-related quality of life, as measured by the global health domain of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30) between baseline and at time of first-restaging scan.
I. To evaluate the prognostic effect of baseline number of circulating tumor cells (CTCs).
II. To determine whether the change in number of CTCs post 2 months of treatment from baseline is associated with PFS and OS.
III. To evaluate the prognostic effect of baseline circulating cell-free deoxyribonucleic acid (cfDNA).
IV. To determine whether the change in cfDNA at 2 months of treatment from baseline is associated with PFS and OS.
V. To compare prostate-specific membrane antigen (PSMA) positron emission tomography/magnetic resonance imaging (PET/MR) or PET/computed tomography (CT) with conventional MRI or CT for evaluation of treatment response.
VI. To correlate drug response in patient derived organoids with clinical response and characterize the tumor microenvironment.
Patients receive futibatinib orally (PO) once daily (QD) on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab intravenously (IV) over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
After completing study treatment, patients are followed up at 30 days, every 9 weeks for up to 18 months, and then every 6 months for up to 5 years.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study of FGFR Inhibitor Futibatinib in Combination With Anti-PD-1 Antibody Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma With FGF19 Expression After First Line Therapy|
|Actual Study Start Date :||May 7, 2021|
|Estimated Primary Completion Date :||May 6, 2023|
|Estimated Study Completion Date :||May 6, 2024|
Experimental: Treatment (futibatinib, pembrolizumab)
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
Other Name: TAS-120
Other: Quality-of-Life Assessment
Other Name: Quality of Life Assessment
- Progression-free survival (PFS) [ Time Frame: At 6 months ]PFS will be calculated using the Kaplan-Meier method.
- Overall response rate (ORR) [ Time Frame: Up to 5 years ]ORR defined as the number of evaluable patients achieving a response (partial response or complete response per Response Evaluation Criteria in Solid Tumors v1.1) during treatment with study therapy divided by the total number of evaluable patients. Point estimates will be generated for objective response rates along with 95% binomial confidence intervals.
- Overall survival (OS) [ Time Frame: Time from registration to death due to any cause, assessed up to 5 years ]The distribution of survival time will be estimated using the method of Kaplan-Meier. OS medians will be estimated along with 95% confidence intervals.
- Incidence of adverse events [ Time Frame: Up to 5 years ]Adverse events will be evaluated, per Common Terminology Criteria for Adverse Events version 5.0, for each patient.
- Change in quality of life (QOL) [ Time Frame: Up to 5 years ]As measured by the global health domain of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire. The median QOL change from baseline along with a 95% confidence interval will be estimated using the Hodges-Lehmann method.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04828486
|United States, Minnesota|
|Mayo Clinic in Rochester||Recruiting|
|Rochester, Minnesota, United States, 55905|
|Contact: Clinical Trials Referral Office 855-776-0015 email@example.com|
|Principal Investigator: Nguyen H. Tran, M.D.|
|Principal Investigator:||Nguyen H Tran||Mayo Clinic in Rochester|