Synergy in Action

Advanced CNS Clinical Trials Course 2021

Jointly sponsored by ISCTM and ASCP
A new, brief, intensive, interactive program for mid-career CNS clinical researchers who wish to expand and refine their trial design expertise

Registration is reserved for accepted applicants, faculty and preceptors: Register here.

Overall course objectives:

During this 1.75-day interactive course, approximately 40 selected students will work closely with CNS trial design and methods experts who represent both academia and industry. Although open to all applicants who meet training prerequisites, this course will build on principles taught at the ASCP Clinical Trials Workshop.  Students will participate in interactive didactic sessions covering elements of early phase clinical trials, including proof-of-mechanism designs, target dosing for first efficacy trials, biomarker validation/interpretation, and the capacity for pre-clinical and biomarker work to reliably predict degree of effect in clinical trials. Later phase trial design topics will include dose-finding strategies, signal detection risks and challenges, and the critical challenge of data interpretation in the context of go/no-go decisions, follow-on grant applications, and publication.  Students will hear from statistical experts in the field who will shed additional light on strategies, controversies, risks, and known regulatory and reviewer/funding requirements in both early and later phase clinical trials. Course content will also address clinical trial design issues which are part of recent NIH RFAs and program announcements. After the didactic sessions, students will break into faculty-assisted small groups to design a protocol, integrating the clinical, relevant psychopharmacological, statistical, and regulatory lessons highlighted.

Faculty and preceptors:
Carla Canuso, MD, Janssen Research and Development
Lori Davis, MD, Tuscaloosa VA Medical Center
Michael Davis, MD, PhD, FDA
Don Goff, MD, NYU School of Medicine
John Kane, MD, The Zucker Hillside Hospital
Bruce Kinon, MD, Cyclerion Therapeutics
Andy Krystal, MD, University of California, San Francisco
Craig Mallinckrodt, PhD, Cortexyme
Kari Nations, PhD, Syneos Health
Elena Polverejan, PhD, Janssen Research and Development
Bill Potter, MD, PhD, Independent expert (retired from NIMH, Lilly, and Merck)
Nina Schooler, PhD, SUNY Downstate Medical Center
Madhukar Trivedi, MD, University of Texas Southwestern Medical School
Daniel Umbricht, MD, xperimed LLC

Agenda at a Glance

Thursday 2 December 2021  
8:00am EST Introduction Carla Canuso
  Early Phase Trials Session 1 – Proof of Mechanism Studies Andy Krystal
  Early Phase Trials Session 2 – Proof of Concept Studies Daniel Umbricht
  Early Phase Statistical Considerations Craig Mallinckrodt
  Pre-Breakout Practical Considerations Kari Nations
  Learning Lab Breakout A: Study Design Group Assignment  
  Late Phase Trials Session 1 – Confirmatory Industry Trials Bruce Kinon
  Late Phase Trials Session 2 – Multi-center Academic Trials Lori Davis
5:15pm EST Session Adjourns  
5:30-7:30pm EST Dine-around with Faculty and Preceptors (optional)
Friday 3 December 2021  
7:00am EST Lite Breakfast with Faculty and Preceptors (optional – lite breakfast provided)  
8:00am EST Late Phase Statistical Considerations  Elena Polverejan
  Learning Lab Breakout B: Study Design Group Assignment  
  Group Discussion of Study Designs  
2:30pm EST Meeting adjourns  

Optional Activities
Thursday: Dine-around with Faculty and Preceptors
Friday: Lite Breakfast with Faculty and Preceptors

Topics such as Adaptive Designs, Regulatory Process, Biomarker Development and Validation, and Designing Multicenter Trials will be discussed during dinner and breakfast.

Registration Fees
Academia/Government: $250
Industry: $500

Who should attend:

  • Academia: Mid-career scientists
  • Government: Mid-career scientists
  • Industry: Associate Director to Senior Director level clinical/medical scientists
  • Clinical: Independent site investigators designing and conducting sponsored or investigator-initiated trials
  • Individuals who have attended the ASCP Clinical Trials Workshop (Clinical Trials in Psychopharmacology) are encouraged to attend this next-level program; however, prior ASCP trials workshop completion is not required, provided the individual meets other training prerequisites listed below

Training prerequisites:

  • MD, PhD (applicable field), PharmD, MS/PhD biostatistics, or MS/PhD epidemiology
  • At least 3 years’ experience designing and executing clinical trials
  • Some prior formalized clinical trial design training and/or supervised design experience mentored by senior trialists
  • Basic level of understanding of applicable inferential statistical principles, power analysis, and data interpretation
  • Working knowledge of neuropharmacology elements relevant to CNS clinical trial

Travel awards: Ten merit and need based travel award stipends will be granted to students seeking support.