July 21-27, 2012
Vail Marriott Mountain Resort & Spa
Vail, Colorado
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Learning Objectives

Clinical Development and Concepts in Biostatistics

  • The sequence of questions in a drug/device clinical development plan
  • Safety monitoring at different stages of development
  • Fundamental concepts in biostatistics and their place in the sequence of clinical development

Phase I and Clinical Pharmacology

  • Goals for Phase I
  • Phase I populations
  • Approaches to define dosage range: Analyses
  • Phase I designs and approaches
  • Use of toxicology studies to shape dosage range and safety surveillance
  • Fundamentals of clinical pharmacology

Phase II, Endpoints, Biomarkers

  • Goals for Phase II
  • Choosing a study population
  • Approaches to choosing clinical endpoints
  • Phase II designs and analyses
  • Use of biomarkers in Phase II

Phase III and Trial Implementation

  • Elements needed for a Phase III trial
  • Stopping/altering studies for safety or efficacy
  • The clinical trial team
  • Data management
  • Realistic timelines, budgets
  • Drug supply
  • Barriers to recruitment
  • Funding and reporting

Investigator Responsibilities, Ethics, and Regulatory

  • GCP and investigator responsibilities, budget
  • Informed consent and risk in early phase trials
  • Regulatory requirements for IND/IDE and reporting requirements

Continuing Medical Education (TBD)

NIH Protocol Template

Protocol Synopsis Template

Clinical Trial Methods Course in Neurology 2012 Agenda

Saturday, July 21, 2012

8:00-1:00 Course Registration, Faculty and Participants register with course staff
12:00-1:00 Faculty Orientation (Lunch)
12:00-1:00 Lunch
1:00-1:15 Introduction to the Course (Kieburtz & Ravina)
1:15-1:30 Review of Materials, expectations, etc
1:30-2:00 Lecture 1: The Changing Landscape for Pharmaceutical Innovation: Economic Challenges in CNS Product Development (Kaitin/Olanow)
2:00-2:45 Lecture 2: Asking Questions: The Sequence of Clinical Development (O’Neill/Katz)
2:45-3:00 Break
3:00-3:45 Lecture 3: Ins/Outs of protocols: language/vocabulary (Blakely/Valadka)
3:45-5:15 Protocol Development 1: Groups Meet (review submitted synopsis; Assign: revise for next session)
5:15-6:00 Opening Reception
6:00-8:00 Group Dinner (Outside weather permitting)

Sunday, July 22, 2012

7:00-8:15 Group Breakfast
8:15-8:45 From Vail to Trial (Erika Augustine/Kieburtz)
8:45-9:30 Lecture 4: Fundamentals of Biostatistics (Parides/Elm)
9:30-10:15 Lecture 5: Questions and Designs for Phase I Clinical Trials (Piantadosi/McDermott)
10:15-10:30 Break
10:30-11:15 Lecture 6: Clinical Pharmacology (Anderson/Venuto)
11:15-12:00 Lecture 7: Goals and Designs for Phase II Clinical Trials (Cudkowicz/Lenz)
12:00-1:00 Lunch
1:00-1:45 Lecture 8: CRM Workshop (Piantadosi)
1:45-4:00 Protocol Development 2: What Is Your Primary Question? (Assign: edit synopsis to submit 3rd session)
4:00-4:15 Break (as needed, snacks)
4:15-5:45 Independent Study/Individual Sessions/Free Time
6:00-7:45 Group Dinner

Monday, July 23, 2012

7:00-8:00 Group Breakfast
8:00-8:45 Lecture 9: Statistical Considerations to Phase II Clinical Trials (McDermott/Kryscio)
8:45-9:30 Lecture 10: Adaptive designs in Phase I-II Clinical Trials (Clarke/McDermott)
9:30-9:45 Break
9:45-10:30 Lecture 11: Endpoints and Outcome Measures (Shefner)
10:30-11:15 Lecture 12: Biomarkers in Clinical Development (Cedarbaum/Olanow)
11:15-12:00 Lecture 13: FDA 101 - What do you need for an IND (Katz/Kieburtz)
12:00-1:00 Lunch (all others)
12:00-1:00 Working lunch (Faculty): update meeting
1:00-1:45 Lecture 14: FDA Regulation of Medical Devices (Como/Valadka)
1:45-4:00 Protocol Development 3: Focus on General Design (review assignment; Assign: revise analysis approach for next session)
3:45-4:15 Break as needed (snacks)
4:00-5:30 Independent study/Free time/sign up to meet with faculty
5:30 Dinner on own

Tuesday, July 24, 2012

7:00-8:00 Group Breakfast
8:00 Updated Protocol Synopsis Due
8:00-8:45 Lecture 15: Safety Monitoring Across Clinical Development (Kieburtz/Haley)
8:45-9:30 Lecture 16: Approaches to Data Monitoring for Safety and Efficacy (Elm/Clarke)
9:30-9:45 Break
9:45-10:30 Lecture 17: Ethical Trials & Neurological Disorders: Intro into Phase I-II Clinical Trials (Kimmelman/Ravina)
10:30-11:15 Lecture 18: Recruitment, Retention and Special Populations (Bell/Cudkowicz)
11:15-12:30 Protocol Development 4: Focus on endpoints and analysis plan
12:30-1:30 Lunch
1:30-4:30 Independent Study/Free time/sign up to meet with faculty Dinner on own
Dinner on own

Wednesday, July 25, 2012

7:00-8:00 Group Breakfast
8:00-9:30 Protocol Development 5: Focus on safety issues and recruitment
9:30-9:45 Break
9:45-10:30 Lecture 19: Clinical Trial Budgets and Practicalities of Operations (Kamp/Lenz)
10:30-11:15 Lecture 20: Understanding the Basics Good Clinical Practices and Investigator Responsibilities (Kayson/Poole)
11:15-11:30 Break
11:30-12:00 Lecture 21: Contracts, Conflicts, and Confidentiality (Shinaman/Kayson)
12:00-12:30 Lecture 22: Attributes of a Good Clinical Trials Site (Cedarbaum/Kamp)
12:30-1:30 Lunch
1:30-4:00 Independent Study (all faculty available)
4:00 Final Protocols Due
Dinner on own

Thursday, July 26, 2012

7:00-8:00 Group Breakfast
8:00-8:30 Lecture 23 A: Funding Opportunities for Clinical Trials: NIH (Moy/Ravina)
8:30-9:00 Lecture 23 B: Funding Opportunities for Clinical Trials: Other Sources (Schwarzschild/Moy)
9:00-9:45 Lecture 24: Are we there yet? What You Need for a Phase III Clinical Trial (Garofalo/Kamp)
9:45-10:00 Break
10:00-11:30 Protocol Development 6: Feedback from Faculty on Protocols, 15 minute sessions will be scheduled with each participant to meet with the faculty of your protocol development team
11:30-12:00 Group photo
12:00-1:00 Lunch
1:00-1:30 Feedback from Trainees
1:30-2:15 Faculty Meeting
2:15-4:15 Participants Complete Course Questionnaires
4:15-4:45 Closing
5:00-6:00 Reception
6:00-8:00 Group Dinner

Friday, July 27, 2012

7:00-8:00 Group Breakfast/Departures


Supported by the National Institute of Neurological Disorders and Stroke NINDS Logo

Jointly Sponsored by:
UR Sponsorship