NCT07069699

Testing the Safety of Anti-Cancer Drug, CX-5461 (Pidnarulex), in Treating Lymphoma With Specific Changes in the MYC Gene

Phase 1b/2 Trial of Pidnarulex in MYC Aberrant Lymphoma

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Burkitt Lymphoma
• Double-Expressor Lymphoma
• High Grade B-Cell Lymphoma With MYC and BCL2 and/or BCL6 Rearrangements

Interventions

Type	Name	Description
PROCEDURE	Biopsy Procedure	Undergo tumor biopsy
PROCEDURE	Biospecimen Collection	Undergo CSF and blood sample collection
PROCEDURE	Computed Tomography	Undergo PET/CT scan
PROCEDURE	Lumbar Puncture	Undergo lumbar puncture
DRUG	Pidnarulex	Given IV
PROCEDURE	Positron Emission Tomography	Undergo PET/CT scan

Timeline

• Start date: 2026-10-13
• Primary completion: 2030-01-31
• Completion: 2030-01-31
• First posted: 2025-07-17
• Last updated: 2026-04-13

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study