NCT04910152

Novel BET Inhibitor PLX51107 for Steroid-Refractory Acute GVHD

A Single Arm, Open Label, Phase 1b/2 Study of Novel BET Inhibitor PLX51107 for Steroid-Refractory Acute GVHD

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Hannah Choe, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase Ib/II trial studies the side effects of PLX51107 in treating steroid-refractory acute graft versus host disease (GVHD). PLX51107 is a novel, potent non-benzodiazepine structured small molecule BET inhibitor with a unique binding mode selective for BRD4 inhibition and a more tolerable side effect profile. PLX51107 may work better in treating steroid-refractory acute GVHD.

Detailed description

PRIMARY OBJECTIVES: I. To evaluate the safety and tolerability of BRD4 inhibitor PLX51107 (PLX51107) as a single agent for allogeneic transplant recipients with steroid-refractory acute graft versus host disease (GVHD). II. To assess the pharmacokinetic (PK) and pharmacodynamic (PD) of orally administered PLX51107 in steroid-refractory acute GVHD patients. SECONDARY OBJECTIVE: I. To evaluate the preliminary efficacy of PLX51107 in steroid-refractory acute GVHD patients. OUTLINE: Patients receive BRD4 inhibitor PLX51107 orally (PO) once daily (QD) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days and then up to 6 months.

Conditions

• Acute Graft Versus Host Disease
• Steroid Refractory Graft Versus Host Disease

Interventions

Type	Name	Description
DRUG	BRD4 Inhibitor PLX51107	Given PO

Timeline

• Start date: 2022-04-19
• Primary completion: 2023-09-28
• Completion: 2023-09-28
• First posted: 2021-06-02
• Last updated: 2025-01-28
• Results posted: 2025-01-28

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study