International Bladder Cancer Group Consensus Statement on Clinical Trial Design for Patients with Bacillus Calmette-Guérin–exposed High-risk Non–muscle-invasive Bladder Cancer

2021 ◽  
Author(s):  
Mathieu Roumiguié ◽  
Ashish M. Kamat ◽  
Trinity J. Bivalacqua ◽  
Seth P. Lerner ◽  
Wassim Kassouf ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bladder Cancer ◽  
Trial Design ◽  
Consensus Statement ◽  
Clinical Trial Design ◽  
Bacillus Calmette Guerin ◽  
Group Consensus ◽  
Muscle Invasive Bladder Cancer ◽  
Cancer Group ◽  
Muscle Invasive

scholarly journals Papillary Recurrence of Bladder Cancer at First Evaluation after Induction Bacillus Calmette-Guérin Therapy: Implication for Clinical Trial Design

2016 ◽  
Vol 70 (5) ◽  
pp. 778-785 ◽  
Author(s):  
Chinedu O. Mmeje ◽  
Charles C. Guo ◽  
Jay B. Shah ◽  
Neema Navai ◽  
H. Barton Grossman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bladder Cancer ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Bacillus Calmette Guerin

scholarly journals Definitions, End Points, and Clinical Trial Designs for Non–Muscle-Invasive Bladder Cancer: Recommendations From the International Bladder Cancer Group

2016 ◽  
Vol 34 (16) ◽  
pp. 1935-1944 ◽  
Author(s):  
Ashish M. Kamat ◽  
Richard J. Sylvester ◽  
Andreas Böhle ◽  
Joan Palou ◽  
Donald L. Lamm ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bladder Cancer ◽  
Sample Size ◽  
Risk Category ◽  
Muscle Invasive Bladder Cancer ◽  
Eligibility Criteria ◽  
End Points ◽  
Cancer Group ◽  
Clinical Trial Designs ◽  
Muscle Invasive

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-Guérin (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 definitions, end points, and clinical trial designs for NMIBC to encourage uniformity among studies in this disease.

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