Precision Metrics for Cognitive and Adaptive Measures in Clinical Trials

Click to view webinar recording of the Q&A session

This webinar session reconvened the expert panel for a follow-up discussion to the recorded presentations below.

There is an urgent need to develop and implement precise metrics for quantitating change in children with lysosomal and other neurological diseases. Growth scale values (GSVs) measure longitudinal cognitive and adaptive changes in early onset and severe neurological diseases. GSV’s are available for tests that are widely used in clinical trials such as the Bayley and the Vineland. 

These presentations were originally recorded at a special session at the February 2022 WORLDSymposium. 

VIDEO LINK: Introduction and insights
Elsa Shapiro, PhD, ABPP (CHAIR)
Shapiro Neuropsychology Consulting, LLC
Professor of Pediatrics and Neurology
University of Minnesota
Portland, OR

VIDEO LINK: Growth scale values (GSVs): Theory, development, and characteristics
Mark H. Daniel, PhD
Mark Daniel Services, LLC
Blaine, MN

VIDEO LINK: Advantages of GSVs in clinical trials
Paul E. Williams, PsyD
Senior Pharma Assessment Consultant
Pearson Clinical Assessment
Orlando, FL

VIDEO LINK: Measuring cognitive outcomes with GSV’s in MPS I
Julie B. Eisengart, PhD, LP
Associate Professor Department of Pediatrics
Director Neurodevelopmental Program in Rare Disease
Division of Clinical Behavioral Neuroscience
University of Minnesota
Minneapolis, MN

VIDEO LINK: Use of GSV scores in natural history of MPS IIIB
Bernice Kuca, MS
Head of Clinical/Regulatory Operations
Allievex Corporation
Boston, MA

VIDEO LINK: Use of GSVs using the Vineland in CLN3 Batten disease
Heather Adams, PhD
Associate Professor
Department of Neurology – Division of Child Neurology
University of Rochester Medical Center
Rochester, NY

VIDEO LINK: Measuring cognitive and developmental outcomes in trials for neuronopathic lysosomal diseases
Patroula Smpokou, MD
Deputy Director
Division of Rare Diseases & Medical Genetics (DRDMG)
Office of New Drugs, CDER, FDA
Washington, DC

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