NCT04890613

Study of CX-5461 in Patients With Solid Tumours and BRCA1/2, PALB2 or Homologous Recombination Deficiency (HRD) Mutation

Phase Ib Expansion Study of CX-5461 in Patients With Solid Tumours and BRCA2 and/or PALB2 Mutation

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 52 (estimated)
• Sponsor: Senhwa Biosciences, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, multi-center, phase 1b study designed to determine a tolerable dose of CX-5461 administered by IV infusion on Day 1 and Day 8 of a 28-day cycle in patients with selected solid tumours and associated mutations for future Phase II trials. The safety and tolerability of CX-5461, preliminary evidence of antitumor effect and the effect of CX-5461 on the Health-Related Quality of Life (HRQoL) will also be evaluated. The study will also evaluate the predictive value of mutational signatures and explore the significance of dynamic changes in ctDNA levels and plasma DNA methylome profiling in this study's exploratory cohort.

Detailed description

CX-5461 (Pidnarulex), a synthetically-derived small molecule that selectively kills HR-deficient cancer cells through the binding and stabilization of G4 DNA structure. Early phase 1 studies suggest CX-5461 has activity and warrants further investigation in HR-deficient tumors. This study aims to determine the tolerable dose of CX-5461 for phase II studies, amongst an expansion cohorts of BRCA1/2, PALB2 or HRD mutant tumors.

Conditions

• Advanced Solid Tumor

Interventions

Type	Name	Description
DRUG	CX-5461	150 mg sterile lyophilized powder containing 1% sucrose

Timeline

• Start date: 2021-09-08
• Primary completion: 2025-12-01
• Completion: 2026-12-01
• First posted: 2021-05-18
• Last updated: 2025-08-13

Locations

8 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study