Reproducibility of cognitive endpoints in clinical trials: lessons from neurofibromatosis type 1
Abstract
Rapid developments in understanding the molecular mechanisms underlying cognitive deficits in neurodevelopmental disorders have increased expectations for targeted, mechanism‐based treatments. However, translation from preclinical models to human clinical trials has proven challenging. Poor reproducibility of cognitive endpoints may provide one explanation for this finding. We examined the suitability of cognitive outcomes for clinical trials in children with neurofibromatosis type 1 (NF1) by examining test‐retest reliability of the measures and the application of data reduction techniques to improve reproducibility.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Dec 01, 2019
- Source ID
- 10.1002/acn3.50952
Entities
People
- Alan B Cantor
- Belinda Barton
- Bruce R. Korf
- Celiane Rey‐casserly
- David Coghill
- David H. Gutmann
- David Viskochil
- Elizabeth K. Schorry
- Gary R. Cutter
- Gérard A. Gioia
- Iris Paltin
- James H. Tonsgard
- Jonathan M Payne
- Joseph D. Ackerson
- Karin S Walsh
- Kathryn North
- Kristina M. Haebich
- Laura Klesse
- Maria T. Acosta
- Michael J. Fisher
- Nicole J Ullrich
- Roger J. Packer
- Stephen Hearps
- Tena Rosser
- The Nf Clinical Trials Consortium
Organizations
- Boston Children's Hospital
- Children's Hospital Los Angeles
- Children's Hospital of Philadelphia
- Children's National Hospital
- Cincinnati Children's Hospital Medical Center
- National Institutes of Health
- Royal Children's Hospital
- The Children's Hospital at Westmead
- United States Department of Defense
- University of Alabama at Birmingham
- University of Chicago
- University of Melbourne
- University of Sydney
- University of Texas at Austin
- University of Utah
- Washington University in St. Louis