Pembrolizumab and GM-CSF in Biliary Cancer
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|ClinicalTrials.gov Identifier: NCT02703714|
Recruitment Status : Active, not recruiting
First Posted : March 9, 2016
Last Update Posted : September 11, 2020
This is an open label phase II trial to examine efficacy and safety of a novel combination of pembrolizumab plus induction GM-CSF in patients with advanced biliary cancers treated at University of California, San Francisco (UCSF).
This phase II study will examine the efficacy and safety of the novel combination of pembrolizumab plus induction GM-CSF in advanced biliary cancer patients with the hypotheses that the combination may increase proportion of patients with overall response compared to contemporary historical controls, with acceptable safety.
|Condition or disease||Intervention/treatment||Phase|
|Biliary Cancer||Drug: Pembrolizumab Drug: Sargramostim||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||42 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Trial of Pembrolizumab (MK-3475) With GM-CSF Induction in Advanced Biliary Cancers|
|Actual Study Start Date :||May 6, 2016|
|Estimated Primary Completion Date :||December 31, 2021|
|Estimated Study Completion Date :||December 31, 2023|
Patients receive pembrolizumab IV over 30 minutes on day 1. Patients also receive sargramostim subcutaneous injection (SC) on days 1-14 of courses 1-2 or 2-3. Treatment repeats every 21 days for up to 2 courses for sargramostim and for up to 35 courses (24 months) for pembrolizumab in the absence of disease or unaccepted toxicity.
200 mg given IV
Other Name: MK-3475
250 µg given SC
Other Name: GM-CSF
- Overall response rate (ORR) [ Time Frame: Response will be assessed from start of treatment through date of discontinuation for up to 2 years ]Proportion of subjects with Response Evaluation Criteria in Solid Tumors (RECIST) 1.1-measurable disease at study entry who have a complete response (CR) or partial response (PR) (confirmed + unconfirmed) using RECIST 1.1 at any time during the main study
- Incidence of adverse events graded using Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: During study treatment and for 30 days after last dose or until start of new treatment (for up to 2 years) ]Safety events will be summarized based on frequency and proportion of total subjects, by system organ class and preferred term. Separate summaries with descriptive statistics will be presented according to anatomic subtype of biliary cancer (e.g. intrahepatic, hilar/perihilar, extrahepatic, gallbladder cancer (GBC)). In overall cohort only related >/=grade 3 Adverse Events (AE)s .
- PD-L1 positive status [ Time Frame: Pre-treatment archival tumor tissue and any tumor tissue sampling required for standard of care during treatment or within 2 years after treatment ]PD-L1 expression will be measured by immunohistochemistry (IHC) and classified as positive or negative by central laboratory testing (QualTek Laboratories) using pre-specified cut-points; will be reported along with 95% confidence interval (CI)
- Progression free-survival [ Time Frame: Within 4 years after start of study treatment ]Time from date of first dose of protocol therapy to date of first documented radiographic and/or clinical disease progression or death from any cause
- Duration of response [ Time Frame: Within 4 years after start of study treatment ]Time from first documented evidence of CR or PR until the first documented sign of disease progression or death
- Overall survival [ Time Frame: Within 4 years after start of treatment ]Time from first dose of protocol therapy to the date of death due to any cause
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): NCT02703714
|United States, California|
|University of California San Francisco|
|San Francisco, California, United States, 94158|
|Principal Investigator:||R. Kate Kelley, M.D.||University of California, San Francisco|