This phase Ib/II trial tests the safety, side effects, best dose and how well giving CX-5461 works for the treatment of patients with B-cell non-Hodgkin lymphoma. CX-5461 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CX-5461 may be safe, tolerable and/or effective in treating patients with B-cell non-Hodgkin lymphoma.
PRIMARY OBJECTIVES: I. To define dose limiting toxicity (DLT). (Phase 1) II. To assess toxicity profile. (Phase 1) III. To determine the recommended phase 2 dose (RP2D). (Phase 1) IV. To perform pharmacokinetic (PK) studies. (Phase 1) V. To examine the effect of CX-5461 (Pidnarulex) on gene expression profile of MYC aberrant lymphomas. (Phase 1) VI. To determine the objective response rate (ORR) (complete response \[CR\] and partial response \[PR\]). (Phase 2) SECONDARY OBJECTIVE: I. To observe and record anti-tumor activity. (Phase 1 and 2) EXPLORATORY OBJECTIVES: I. To determine the mechanisms of response and resistance using circulating tumor deoxyribonucleic acid (ctDNA). (Phase 1 and 2) II. To observe cerebral spinal fluid (CSF) distribution of CX-5461 (Pidnarulex). (Phase 1 and 2) OUTLINE: This is a phase 1 dose-escalation study of CX-5461 followed by a phase 2 study. Patients receive CX-5461 intravenously (IV) over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo lumbar puncture with cerebrospinal fluid (CSF) collection on study and positron emission tomography (PET) scan/computed tomography (CT) scan, tumor biopsy, and blood sample collection throughout the study. After completion of study treatment, patients are followed up at 30 days, every 3-4 months for the first 2 years, and every 6 months for years 3-5.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Undergo tumor biopsy
Undergo CSF and blood sample collection
Undergo PET/CT scan
Undergo lumbar puncture
Given IV
Undergo PET/CT scan
Yale University Cancer Center LAO
New Haven, Connecticut, United States
RECRUITINGIncidence of adverse events (Phase 1)
Assessed by the Common Terminology Criteria for Adverse Events version 5. Safety will be summarized overall, by actual dose level in Phase 1b.
Time frame: Up to 30 days after last dose of study treatment
Recommended phase 2 dose (RP2D) (Phase 1)
Time frame: Up to 5 years
Pharmacokinetic (PK) levels (Phase 1)
Assessed using Liquid Chromatography Mass Spectrometry (LC-MS).
Time frame: At cycle (C) 1 day (D) 8 at pre-dose, end of infusion, and 2 hours (hrs) post-dose; on C1D9 at 24 hrs post C1D8 infusion; on C1D10 at 48 hrs post C1D8 infusion; on C1D11 at 72 hrs post C1D8 infusion; and on C1D15 at 167 hrs post C1D8 infusion
Gene expression (Phase 1)
Assessed by ribonucleic acid sequencing complemented by whole exome sequencing using an average absolute log2 fold change relative to baseline with a significance threshold of p \< 0.05.
Time frame: At baseline, C1D9 or C1D10 (within 24-48 hrs of C1D8 dosing),and at progression
Overall response rate (Phase 2)
Assessed using standard radiographic criteria according to Lugano criteria. Phase 2 results will be reported at the RP2D.
Time frame: Up to 5 years
Response to therapy (Phase 1 and 2)
Assessed according to Lugano criteria.
Time frame: Up to 5 years
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