Efficacy of Padeliporfin Vascular-Targeted Photodynamic Therapy for Low-Grade UTUC

The phase 3 ENLIGHTED trial from Jonathan Coleman, MD, and colleagues sought to determine the safety and efficacy of padeliporfin vascular-targeted photodynamic therapy (VTP) for patients with low-grade upper tract urothelial carcinoma (UTUC).

The study was presented at the 2024 American Urological Association Annual Meeting.

Researchers noted that padeliporfin VTP has previously shown positive safety and efficacy results for patients with low-grade UTUC.

In the open-label trial, patients underwent a 2-phase treatment regimen, which included an induction phase and a maintenance treatment phase (MTP). During the induction phase, patients received 1 to 3 VTPs across 4-week intervals until they reached complete response (CR) or treatment failure. Investigators classified patients with high-grade recurrences as treatment failures and removed them from the study.

Patients who achieved CR continued to the MTP and received VTP for recurrent tumors and endoscopic evaluation every 3 months for up to 12 months. Researchers continued to monitor patients who finished the MTP for 48 months to observe long-term outcomes.

The primary end point was CR on endoscopic evaluation and selective cytology upon primary response evaluation during padeliporfin VTP induction.

Of the 12 patients who underwent treatment as of October 2023, 9 finished induction, with 6 (67%) of those patients reaching CR and 3 (33%) reaching primary response.

The most reported treatment-related adverse events (TRAEs) included grade 1 to 2 flank pain (36%), vomiting (15%), nausea (9%), and hematuria (9%), which all patients reported as resolved within a few days. Six percent of patients reported grade 3 TRAEs, such as flank pain, hypertension, and renal colic, which were resolved within 2 days.

“Padeliporfin VTP has shown evidence of safety and efficacy with preliminary data that are consistent with prior experience,” the researchers wrote. “Recruitment for the ENLIGHTED trial is ongoing, with results expected to provide the basis for approval of a new therapy that clinically benefits patients.”