Twenty-two patients were enrolled in the MitoGel UTUC Compassionate Use Program, of which 14 completed treatment. These patients had tumors that were difficult to resect or had extensive tumor burden. Of patients with low grade disease, 8 had complete response and 5 had a partial response. Feasibility and safety was demonstrated.
This lead to a prospective phase 3, open label single arm trial in patients that is currently ongoing. Patients with low grade UTUC are being studied to evaluate the efficacy and safety MitoGel instillation. Patients are given 6 once weekly instillations of MitoGel (4mg/1ml) with 1.3gm of Sodium Bicarbonate the evening prior, morning of and 30 minutes prior to treatment. Seventy-four patients with pathologically confirmed low grade biopsy proven non-invasive disease are needed to complete the trial with a 90% power to demonstrate the observed complete response rate superior to 15%. Tumors may be treatment naïve or recurrent, located at the renal pelvis, infundibula, and or calyces and deemed appropriate for conservative renal sparing endoscopic management. Exclusion criteria includes any history of CIS, high grade disease within the last 2 years, BCG therapy within 6 months, history of invasive disease within the last 5 years, or current treatment with systemic chemotherapy. The primary and secondary efficacy end points include tumor ablative effect at 3 months and its durability at 12 months, respectively. As an update, there are 27 open trial sites with a goal of 45 sites. The first patients were enrolled in April of 2017.
Dr. Lerner looks forward to the results of the data, to hopefully support MitoGel for organ conservation chemoablation therapy for low grade upper tract urothelial carcinoma.
Presented by: Seth P. Lerner, MD. Professor Urology, Baylor College of Medicine, Houston, TX, US Beth and Dave Swalm Chair in Urologic Oncology, Director of Urologic Oncology, Director of the Multidisciplinary Bladder Cancer Program, Faculty Group Practice Medical Director
Written by: David B. Cahn, DO, MBS, @dbcahn, Fox Chase Cancer Center, Philadelphia, PA at the 18th Annual Meeting of the Society of Urologic Oncology, November 20-December 1, 2017 – Washington, DC