1650 PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MYCOBACTERIAL CELL WALL-DNA COMPLEX IN THE TREATMENT OF PATIENTS WITH NON-MUSCLE INVASIVE BLADDER CANCER AT HIGH RISK OF PROGRESSION AND WHO ARE REFRACTORY TO BCG (2011)

You have accessJournal of UrologyBladder Cancer: Superficial1 Apr 20111650 PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MYCOBACTERIAL CELL WALL-DNA COMPLEX IN THE TREATMENT OF PATIENTS WITH NON-MUSCLE INVASIVE BLADDER CANCER AT HIGH RISK OF PROGRESSION AND WHO ARE REFRACTORY TO BCG Alvaro Morales, Harry Herr, Ashish Kamat, Gary Steinberg, Robert Given, Christine Lihou, and Zvi Cohen Alvaro MoralesAlvaro Morales Kingston, Canada More articles by this author , Harry HerrHarry Herr New York, NY More articles by this author , Ashish KamatAshish Kamat Houston, TX More articles by this author , Gary SteinbergGary Steinberg Chicago, IL More articles by this author , Robert GivenRobert Given Norfolk, VA More articles by this author , Christine LihouChristine Lihou Chadds Ford, PA More articles by this author , and Zvi CohenZvi Cohen Belleville, Canada More articles by this author View All Author Informationhttps://doi.org/10.1016/j.juro.2011.02.1760AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookTwitterLinked InEmail INTRODUCTION AND OBJECTIVES The response to second-line therapies in patients whose tumors are refractory to BCG is generally poor. While radical cystectomy is the standard of care following BCG failure, not all patients are suitable candidates. The objective of this open-label, single-treatment arm study was to evaluate the efficacy and safety of intravesical mycobacterial cell wall-DNA complex (MCC) in patients with non-muscle invasive bladder cancer (NMIBC) who were refractory to BCG therapy and at high risk of progression. METHODS Patients with high grade urothelial carcinoma (papillary NMIBC and/or carcinoma in situ [CIS]) who failed to respond to one or more courses of BCG were enrolled. The treatment regimen consisted of 6 weekly intravesical instillations of 8 mg MCC followed by 3 once-weekly instillations at 3, 6, 12, 18 and 24 months. Clinical efficacy assessments included cystoscopy, urine cytology and biopsy. Safety variables included patient reported adverse events (AEs) and serious adverse events (SAEs), vital signs and clinical laboratory assessments. The primary efficacy endpoint was the disease-free survival (DFS) rate at one year based on the intent-to-treat population and was determined using Kaplan-Meier estimates. Patients were considered disease-free when absence of recurrent bladder cancer was confirmed on biopsy by central laboratory histopathology. RESULTS A total of 129 patients were enrolled from 25 centers in US and Canada. The median age was 71 years (range of 41–90). All 129 patients were white and 95 (73.6%) were male. At study entry, 91 (70.5%) patients had CIS, either primary or concomitant with papillary tumors, and 38 (29.5%) had only papillary tumors. All papillary tumors were resected prior to start of treatment. Based on a preliminary analysis, the overall one-year DFS rate was 25.0% with a median DFS interval of 177 days. Sub-analysis showed that patients with CIS tumors on entry had a one-year DFS rate of 21.0%, while patients with only papillary tumors on entry had a one-year DFS rate of 35.1%. Intravesical administration of MCC was well tolerated - most AEs were mild to moderate in severity and few lead to discontinuation of treatment. Two SAEs (hematuria, urinary tract infection) were considered as being possibly related to the investigational treatment. CONCLUSIONS The preliminary results indicate that MCC has activity in patients with BCG-refractory NMIBC and may provide an alternative to cystectomy in this population. © 2011 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 185Issue 4SApril 2011Page: e662-e663 Advertisement Copyright & Permissions© 2011 by American Urological Association Education and Research, Inc.MetricsAuthor Information Alvaro Morales Kingston, Canada More articles by this author Harry Herr New York, NY More articles by this author Ashish Kamat Houston, TX More articles by this author Gary Steinberg Chicago, IL More articles by this author Robert Given Norfolk, VA More articles by this author Christine Lihou Chadds Ford, PA More articles by this author Zvi Cohen Belleville, Canada More articles by this author Expand All Advertisement Advertisement PDF downloadLoading ...