Design, Execution and Oversight of Multicenter RCTs for Children with Low-Frequency Hematologic and Vascular Diseases: Knowledge Gained and Lessons Learned from Kids-DOTT

Grand Rounds
Conference Date 
Wednesday, October 12, 2022 - 12:00 to 13:00
Name of speaker 
Dr. Neil Goldenberg
Description 

Dr. Neil Goldenberg, MDCM, PhD
Johns Hopkins University School of Medicine,
Johns Hopkins All Children’s Hospital & JHAC Institute of Clinical and Translational Research

Objectives 
  1. To appreciate advantages of the parallel-cohort RCT design as applied to rare diseases/low-frequency conditions.
  2. To understand the principles underlying the bivariate endpoint analytic method and its suitability for modeling efficacy-safety trade-off in phase 3 antithrombotic trials.
  3. To become familiar (or refresh familiarity) with endpoint quality assurance measures that are critical to maximizing “signal-to-noise” in clinical trials.
  4. To appreciate the findings and clinical practice implications of the Kids-DOTT multinational randomized clinical trial on duration of anticoagulation for venous thromboembolism treatment in patients <21 years old.

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