This phase I/II trial studies the side effects and best dose of pidnarulex when given together with cemiplimab and to see how well it works in treating patients with microsatellite stable (MSS) colorectal cancer (CRC) that does not respond to treatment (refractory). Pidnarulex may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving pidnarulex with cemiplimab may be safe, tolerable and/or effective in treating patients with refractory MSS CRC.
PRIMARY OBJECTIVES: I. To establish the recommended phase 2 dose of pidnarulex (CX-5461) with anti-programmed cell death protein 1 (PD-1) in phase 1, and to determine safety and tolerability of pidnarulex (CX-5461) alone, and in combination with anti-PD-1 in phases 1 and 2. II. To determine the progression-free survival (PFS) of pidnarulex (CX-5461) alone and in combination with anti-PD-1 in patients with refractory liver metastatic microsatellite stable (MSS) colorectal cancer (CRC) associated with replication stress in phase 2. SECONDARY OBJECTIVES: I. To observe and record anti-tumor activity. II. To compare the objective response rate (ORR) and disease control rate (DCR) of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. III. To compare the duration of response (DoR) of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. IV. To compare the overall survival (OS) of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. V. To evaluate plasma pharmacokinetic (PK) profiles of pidnarulex (CX-5461) alone and in combination with anti-PD-1. VI. To evaluate the plasma PK profile of cemiplimab. VII. To explore gene signature patterns at baseline or following treatment that may suggest, response to pidnarulex (CX-5461) alone or in combination with anti-PD-1, by whole exome sequencing (WES) and ribonucleic acid (RNA) sequencing in tumor tissue and cell-free deoxyribonucleic acid (DNA) in peripheral blood in phase 2. EXPLORATORY OBJECTIVES: I. To evaluate the baseline expression of MYC and CCNE1 in tumor tissue and its association with response to treatment, as identified by immunohistochemistry in phase 2. II. To evaluate the stimulator of interferon genes (STING) pathway activation and immune cell profile in the tumor at baseline and after treatment with pidnarulex (CX-5461) alone or in combination with anti-PD-1, and its association with response to treatment, as identified by immunohistochemistry in phase 2. III. To evaluate replication stress at baseline and after treatment, and its association with response to treatment, as identified by immunohistochemistry in phase 2. IV. To explore pidnarulex (CX-5461) target engagement, as identified by 47S pre-ribosomal ribonucleic acid (rRNA) in-situ hybridization in phase 2. V. To explore DNA alterations in circulating-tumor deoxyribonucleic acid (ctDNA) and their potential correlation with response to treatment in phase 2. OUTLINE: This is a phase I, dose-escalation study of pidnarulex in combination with cemiplimab followed by a phase II study. PHASE I: Patients receive cemiplimab intravenously (IV) over 30 minutes on days 1 and 15 of each cycle and pidnarulex IV over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection on study and computed tomography (CT), magnetic resonance imaging (MRI), or positron emission tomography (PET)/CT throughout the trial. PHASE II: Patients are randomized to 1 of 2 arms. ARM I: Patients receive pidnarulex IV over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo tumor biopsy and CT, MRI, or PET/CT throughout the trial. Additionally, patients undergo blood sample collection on study. ARM II: Patients receive cemiplimab IV over 30 minutes on days 1 and 15 of each cycle and pidnarulex IV over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo tumor biopsy and CT, MRI, or PET/CT throughout the trial. Additionally, patients undergo blood sample collection on study. After completion of study treatment, patients are followed up at 30 days and then every 3 months for 2 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
86
Undergo tumor biopsy
Undergo blood sample collection
Given IV
Undergo CT or PET/CT
Undergo MRI
Given IV
Undergo PET/CT
UCHealth University of Colorado Hospital
Aurora, Colorado, United States
RECRUITINGMedStar Georgetown University Hospital
Washington D.C., District of Columbia, United States
RECRUITINGEmory University Hospital Midtown
Atlanta, Georgia, United States
RECRUITINGEmory University Hospital/Winship Cancer Institute
Atlanta, Georgia, United States
RECRUITINGEmory Saint Joseph's Hospital
Atlanta, Georgia, United States
RECRUITINGUniversity of Maryland/Greenebaum Cancer Center
Baltimore, Maryland, United States
RECRUITINGJohns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States
RECRUITINGRecommended phase 2 dose (RP2D) of pidnarulex (CX-5461) with anti-programmed cell death protein 1 (anti-PD-1) (Phase 1)
The Bayesian optimal interval design will be employed to identify the RP2D of pidnarulex (CX-5461) in combination with anti-PD-1.
Time frame: Up to 28 days
Incidence of adverse events, serious adverse events, and dose limiting toxicities
Will evaluate safety and tolerability of pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phases 1-2. Safety and tolerability will be summarized via descriptive statistics, including adverse events, serious adverse events, and dose limiting toxicities.
Time frame: Up to 30 days after last dose of study drug
Progression-free survival (PFS) (Phase II)
Will evaluate PFS of pidnarulex (CX-5461) alone and in combination with anti-PD-1 in patients with refractory liver metastatic microsatellite stable colorectal cancer associated with replication stress in phase 2. PFS will be compared between the 2 treatment groups following the futility boundary on hazard ratio (HR) derived by using a Hwang-Shih-DeCani spending function with gamma = -2.909. The Kaplan-Meier method will be used to estimate median PFS, PFS rates at different time points, and their 95% confidence intervals (CIs), using the Brookmeyer Crowley's method. The HR and the corresponding two-sided 95% CI will be estimated in a stratified Cox regression model. Stratification factors are consistent with those used at randomization. Subgroup analysis will be performed to assess the consistency of treatment effect across subgroups.
Time frame: From randomization until disease progression (as assessed by Response Evaluation Criteria in Solid Tumors [RECIST] version [v] 1.1) or death from any cause, whichever occurs first, assessed up to 2 years
Objective response rate (ORR) (Phase 2)
Will evaluate the ORR of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. Defined as the proportion (%) of patients with confirmed complete response (CR) or partial response (PR) as assessed according to RECIST v1.1. ORR will be compared to historical control following Simon's two stage design for the within-arm analysis. The cumulative frequency estimated ORR with corresponding 95% CI will be reported.
Time frame: Up to disease progression or subsequent anticancer therapy, or until the last evaluable tumor assessment in the absence of disease progression, assessed up to 2 years
Disease control rate (DCR) (Phase 2)
Will evaluate the DCR of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2.Defined as the proportion (%) of patients with confirmed CR, PR or stable disease as assessed according to RECIST v1.1. The cumulative frequency estimated DCR with corresponding 95% CI will be reported.
Time frame: Up to disease progression or subsequent anticancer therapy, or until the last evaluable tumor assessment in the absence of disease progression, assessed up to 2 years
Duration of response (DOR) (Phase 2)
Will evaluate the DOR of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. Will be evaluated only for patients who achieve CR or PR. DOR will be summarized descriptively using the Kaplan-Meier method.
Time frame: From the first recorded response (confirmed CR or PR) to the first recorded disease progression (according to RECIST v1.1) or death from any cause, whichever occurs first, assessed up to 2 years
Overall survival (OS) (Phase 2)
Will evaluate the OS of patients treated with pidnarulex (CX-5461) alone and in combination with anti-PD-1 in phase 2. OS will be summarized descriptively using the Kaplan-Meier method.
Time frame: From randomization until death from any cause, assessed up to 2 years
Pharmacokinetic (PK) parameters of pidnarulex (CX-5461)
Will evaluate the PK parameters of pidnarulex (CX-5461) when given alone and in combination with anti-PD-1, including volume of distribution, area under the curve, maximum serum concentration, serum half-life, and accumulation. Will be reported descriptively. Will be analyzed with both non-compartmental and nonlinear mixed effects approaches. Will also be compared between the randomized arms.
Time frame: Cycle (C) 1 day (D) 1, C1D8, C1D9, C1D15, C2D1, C2D15, C3D1, C6D1, C9D1, C12D1
PK parameters of cemiplimab (REGN2810)
Will evaluate the PK parameters of cemiplimab (REGN2810), including clearance. Will be reported descriptively. Will be analyzed with both non-compartmental and nonlinear mixed effects approaches.
Time frame: C1D1, C1D8, C1D9, C1D15, C2D1, C2D15, C3D1, C6D1, C9D1, C12D1
Gene signature patterns (Phase 2)
Will evaluate gene signature patterns at baseline or following treatment that may suggest response to pidnarulex (CX-5461) alone or in combination with anti-PD-1, by whole exome sequencing and ribonucleic acid sequencing in tumor tissue and cell-free deoxyribonucleic acid (DNA) in peripheral blood in phase 2.
Time frame: At baseline and 30 days after last dose of study drug
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