RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:A merger between TST and TLT? Not likely (final edition) Below is the trial design and acceptable Efficacy for each stage, Dr. Kamats presentation on mcna was to review results and conclude that the efficacy and trial design are acceptable. Based on their evalaution 94 patiemts out of the 129 patient trial 301 fall under the non-responsive category.
Definitions, End Points, and Clinical Trial Designs for Non–Muscle-Invasive Bladder Cancer: Recommendations From the International Bladder Cancer Group Ashish M. Kamat, Richard J. Sylvester, Andreas Bohle, Joan Palou, Donald L. Lamm, Maurizio Brausi, ¨ Mark Soloway, Raj Persad, Roger Buckley, Marc Colombel, and J. Alfred Witjes Listen to the podcast by Dr Galsky at www.jco.org/podcasts ABSTRACT Purpose To provide recommendations on appropriate clinical trial designs in non–muscle-invasive bladder cancer (NMIBC) based on current literature and
expert consensus of the International Bladder Cancer Group. Methods We reviewed published trials, guidelines, meta-analyses, and reviews and provided recommendations on eligibility criteria, baseline evaluations, end points, study designs, comparators, clinically meaningful magnitude of effect, and sample size. Results NMIBC trials must be designed to provide the most clinically relevant data for the specific risk category of interest (low, intermediate, or high). Specific eligibility criteria and baseline evaluations depend on the risk category being studied. For the population of patients for whom bacillus Calmette-Guerin (BCG) has failed, ´ the type of failure (BCG unresponsive, refractory, relapsing, or intolerant) should be clearly defined to make comparisons across trials feasible. Single-arm designs may be relevant for the BCG-unresponsive population. Here, a clinically meaningful initial complete response rate (for carcinoma in situ) or recurrence-free rate (for papillary tumors) of at least 50% at 6 months, 30% at 12 months, and 25% at 18 months is recommended. For other risk levels, randomized superiority trial designs are recommended; noninferiority trials are to be used sparingly given the large sample size required. Placebo control is considered unethical for all intermediate- and high-risk strata; therefore, control arms should comprise the current guideline-recommended standard of care for the respective risk level. In general, trials should use time to recurrence or recurrence-free survival as the primary end point and time to progression, toxicity, disease-specific survival, and overall survival as potential secondary end points. Realistic efficacy thresholds should be set to ensure that novel therapies receive due review by regulatory bodies. Conclusion The International Bladder Cancer Group has developed formal recommendations regarding defi- nitions, end points, and clinical trial designs for NMIBC to encourage uniformity among studies in this disease. J Clin Oncol 34. © 2016 by American Society of Clinical Oncology INTRODUCTION There is a significant unmet need for new therapies in non–muscle-invasive bladder cancer (NMIBC), as evidenced by the fact that in more than 30 years, only three drugs have been approved by the US Food and Drug Administration (FDA) or European Medicines Agency for treatment of the disease: thiotepa (1959), bacillus Calmette-Guerin (BCG; 1990), and ´ valrubicin (1998). Studies in NMIBC are hampered by lack of consensus on trial end points and appropriate control arms among regulatory bodies and confusion resulting from perceived difficulties related to these factors. In recent years, the American Urological Association (AUA), FDA, European Association of Urology, and others have tried to address these issues and proposed trial designs to support the development of new therapies for NMIBC. Recommendations put forth have been based
Efficacy of Mycobacterium phlei Cell Wall-Nucleic Acid Complex (MCNA) in BCG- Unresponsive Patients By: Ashish K.2, Amrhein J.3, Cohen Z.1, Champagne M.1
Institutes: 1Telesta Therapeutics, Inc, Department, Pointe Claire, Canada, 2The University of Texas M.D. Anderson Cancer Center, Dept. of Urology, Houston, United States of America, 3McDougall Scientific Ltd, Department, Toronto, Canada
Aims and objectives of this presentation To present results of MCNA in patients with BCG Unresponsive NMIBC showing how in this highest risk subgroup of BCG Failures MCNA achieves 1 year DFS of 35% for overall population; 24% for CIS; and 60% for papillary tumors.