Article Information
Group Information: A list of the GWPCARE2 Study Group members appear at the end of the article.
Accepted for Publication: January 9, 2020.
Published Online: March 2, 2020. doi:10.1001/jamaneurol.2020.0073
Correction: This article was corrected on May 11, 2020, to fix the spelling of collaborator Katherine
Moretz, MD’s name, which had been incorrectly given as Katherine Mortez in the Group
Members section.
Open Access: This is an open access article distributed under the terms of the
CC-BY-NC-ND License. © 2020 Miller I et al.
JAMA Neurology.
Corresponding Authors: Ian Miller, MD, Department of Neurology, Nicklaus Children’s Hospital, 3200 SW 60th
Ct, Ste 302, Miami, FL 33155 (
ian.miller@
123456mchdocs.com
); Ingrid E. Scheffer, MBBS, PhD, FRS, Epilepsy Research Centre, 245 Burgundy St,
Heidelberg, Victoria 3084, Australia (
i.scheffer@
123456unimelb.edu.au
).
Author Contributions: Mr Checketts had full access to all of the data in the study and takes responsibility
for the integrity of the data and the accuracy of the data analysis.
Concept and design: Miller, Gil-Nagel.
Acquisition, analysis, or interpretation of data: All authors.
Drafting of the manuscript: Miller, Saneto, Dunayevich, Knappertz.
Critical revision of the manuscript for important intellectual content: All authors.
Statistical analysis: Checketts.
Administrative, technical, or material support: Scheffer, Perry, Dunayevich.
Supervision: Miller, Scheffer, Gunning, Sanchez-Carpintero, Gil-Nagel, Perry, Dunayevich, Knappertz.
Conflict of Interest Disclosures: Dr Miller reported receiving grants and other compensation from Greenwich Biosciences
during the conduct of the study and grants, personal fees, and other compensation
from Greenwich Biosciences, nonfinancial support and other compensation from Dravet
Syndrome Foundation, grants and nonfinancial support from Insys Pharmaceuticals, personal
fees and nonfinancial support from BioMarin Pharmaceutical, Inc, and Upshire-Smith
Laboratories, LLC, and grants, personal fees, and nonfinancial support from Zogenix,
Inc, outside the submitted work. Dr Scheffer reported receiving other compensation
from GW Pharmaceuticals during the conduct of the study; other from Zynerba Pharmaceuticals,
Inc, Zogenix, Inc, Ovid Therapeutics, Inc, Marinus, and Ultragenyx Pharmaceutical,
Inc, personal fees and other compensation from UCB, personal fees from GlaxoSmithKline,
Eisai Co, Ltd, BioMarin Pharmaceutical, Inc, Nutricia, and Xenon Pharmaceuticals,
outside the submitted work; a patent to WO06/133508 issued; and research funding from
the National Health and Medical Research Council, Health Research Council of New Zealand,
and National Institutes of Health. Dr Gunning reported serving on scientific advisory
boards of GW Pharmaceuticals, Zogenix, Inc, and Ovid Therapeutics, Inc/Takeda Pharmaceutical
Company Limited; serving on the speaker’s bureau for LivaNova plc; and serving as
the principal investigator for trials sponsored by GW Research, Ltd, and Zogenix,
Inc. Dr Sanchez-Carpintero reported receiving research support from GW Pharmaceuticals
and Zogenix for clinical trials; serving on advisory boards for Novartis International
AG, GW Pharmaceuticals, and Zogenix, Inc; and serving as a trial investigator for
GW Research, Ltd and Ovid Therapeutics, Inc/Takeda Pharmaceutical Company Limited.
Dr Gil-Nagel reported serving as an investigator, speaker, and advisory board member
for Bial, Eisai Co, Ltd, Esteve, UCB, GW Pharmaceuticals, GW Research, Ltd, Zogenix,
Inc, Stoke Therapeutics, Sanofi, and Arvelle Therapeutics. Dr Perry reported participating
on advisory boards for Upsher-Smith Laboratories, LLC, and Zogenix, Inc, as a consultant
for Encoded Therapeutics, Inc, and as a trial investigator for GW Research, Ltd, and
Zogenix, Inc. Dr Saneto reported serving as a principal investigator on the GW Research,
Ltd, trials for Dravet Syndrome Foundation and TSC; on the data safety monitoring
board for a clinical study sponsored by Reata Pharmaceuticals for Friedreich ataxia;
as principal investigator on a study sponsored by Stealth Pharmaceutical for primary
mitochondrial myopathy; as principal investigator on a study sponsored by Zogenix,
Inc, in patients with Dravet syndrome; and on the speaker’s bureau for GW Pharmaceuticals.
No other disclosures were reported.
Funding/Support: This study was supported by GW Research, Ltd.
Role of the Funder/Sponsor: The sponsor was responsible for the design and conduct of the study. The sponsor
was involved with the collection, management, analysis, and interpretation of the
data; preparation, review, or approval of the manuscript; and decision to submit the
manuscript for publication.
Group Members: The following investigators are members of the GWPCARE2 Study Group: Gregory Barnes,
MD, University of Louisville Autism Center, Louisville, Kentucky; Hari Bhathal, MD,
Neurocenter Barcelona, Centro Médico Teknon, Barcelona, Spain; Verónica Cantarín-Extremera,
MD, Neuropediatrics, Hospital Universitario Niño Jesús, Madrid, Spain; Michael G.
Chez, MD, Sutter Medical Group, Neuroscience, Roseville, California; Dave F. Clarke,
MD, Dell Medical School at University of Texas, Austin; Aviva Fattal-Valevski, MD,
Department of Pediatric Neurology, Dana-Dwek Children’s Hospital, Tel-Aviv Sourasky
Medical Center, Tel Aviv, Israel; Michael Frost, MD, Minnesota Epilepsy Group, St
Paul; Antonio Gil-Nagel, MD, PhD, Hospital Ruber Internacional, Madrid, Spain; Monisha
Goyal, MD, Department of Pediatrics, University of Alabama, Birmingham; Robert S.
Greenwood, MD, Department of Neurology, University of North Carolina School of Medicine,
Chapel Hill; Boudewijn Gunning, MD, Stichting Epilepsie Instellingen Nederland, Zwolle,
the Netherlands; Jonathan J. Halford, MD, Medical University of South Carolina, Charleston;
Julián Lara Herguedas, MD, Neuropediatrics Unit, Pediatrics Unit, Puerta de Hierro,
Majadahonda Hospital, Madrid, Spain; Gregory L. Holmes, MD, Department of Neurological
Sciences, University of Vermont College of Medicine, Burlington; Siddharth Kapoor,
MD, University of Kentucky College of Medicine, Lexington; Katarzyna Kotulska, MD,
The Children’s Memorial Health Institute, Warsaw, Poland; Linda Laux, MD, Ann & Robert
H. Lurie Children’s Hospital of Chicago and Northwestern Feinberg School of Medicine,
Chicago, Illinois; Annick Laridon, MD, Epilepsy Centre for Children and Youth Pulderbos,
Zanhoven, Belgium; John A. Lawson, MBBS, Sydney Childrens Hospital, Randwick, Syndney,
Australia; Paul D. Lyons, MD, Virginia Comprehensive Epilepsy Program at Winchester
Medical School, Winchester; Jennifer Madan Cohen, MD, Connecticut Children’s Medical
Center and University of Connecticut School of Medicine, Hartford; Deepak Madhavan,
MD, Boys Town National Research Hospital, Boys Town, Nebraska; Eric D. Marsh, MD,
Departments of Neurology and Pediatrics, Children’s Hospital of Philadelphia, Perelman
School of Medicine at the University of Pennsylvania, Philadelphia; Maria Mazurkiewicz-Beldzinska,
MD, Medical University of Gdańsk, Gdańsk, Poland; Ian Miller, MD, Nicklaus Children’s
Hospital, Miami, Florida; Wendy G. Mitchell, MD, Children’s Hospital of Los Angeles,
Los Angeles, California; Krystyna Mitosek-Szewczyk, MD, Department of Pediatric Neurology,
Medical University of Lublin, Lublin, Poland; David B. Moore, MD, McFarland Clinic,
Ames, Iowa; Richard P. Morse, MD, Department of Neurology and Pediatrics, Children’s
Hospital at Dartmouth-Hitchcock, Lebanon, New Hampshire; Katherine Moretz, MD, Memorial
Health University Medical Center, Savannah, Georgia; Lawrence Morton, MD, Children’s
Hospital of Richmond at Virginia Commonwealth University, Richmond, Virginia; M. Scott
Perry, MD, Cook Children’s Medical Center, Fort Worth, Texas; Lukasz Przyslo, MD,
PhD, Department of Neurology, Research Institute of Polish Mother’s Memorial Hospital,
Lodz, Poland; Colin Roberts, MD, Doernbecher Childhood Epilepsy Program, Oregon Health
and Science University, Portland; William E. Rosenfeld, MD, The Comprehensive Epilepsy
Care Center for Children and Adults, St Louis, Missouri; Russell P. Saneto, DO, PhD,
Seattle Children’s Hospital/University of Washington and the Neuroscience Institute,
Seattle; Rocio Sanchez-Carpintero, MD, Clínica Universidad de Navarra, Pediatric Neurology
Unit and IdiSNA, Navarra Institute for Health Research, Pamplona, Spain; Ingrid E.
Scheffer, MBBS, PhD, FRS, University of Melbourne, Austin Health, The Royal Children’s
Hospital, Florey Institute and Murdoch Children’s Research Institute, Melbourne, Australia;
Eric Segal, MD, Hackensack University Medical Center, Hackensack Meridian School of
Medicine, Hackensack, New Jersey; Gregory B. Sharp, MD, Arkansas Children’s Hospital/University
of Arkansas for Medical Sciences, Little Rock; Juan R. Uranga, MD, Clínica Plaza de
las Monjas, Huelva, Spain; Vicente Villanueva, MD, Refractory Epilepsy Unit, Neurology
Service, Hospital Universitario y Politécnico La Fe, Valencia, Spain; Arie Weinstock,
MD, Jacobs School of Medicine and Biomedical Sciences, Oishei Children’s Outpatient
Center, Buffalo, New York; Bruria Ben Zeev, MD, Department of Pediatric Neurology,
Dana-Dwek Children’s Hospital Tel-Aviv Sourasky Medical Center, Tel Aviv, Israel;
and Marta Żołnowska, MD, Centrum Medyczne Plejady, Kraków, Poland.
Meeting Presentations: Data from this study were previously presented at the American Academy of Neurology
annual meeting; May 7, 2019; Philadelphia, Pennsylvania; the 33rd International Epilepsy
Congress; June 24, 2019; Bankok, Thailand; and the Child Neurology Society annual
meeting; October 24 and 25, 2019; Charlotte, North Carolina.
Additional Contributions: We thank the patients, their families, and the study sites that participated in this
trial. Scott Polen, BA, Greenwich Biosciences, Inc, contributed as the clinical study
manager; Kevan VanLandingham, MD, PhD, Greenwich Biosciences, Inc, contributed to
the study design and interpretation of the data; Kenneth Sommerville, MD, PhD, Greenwich
Biosciences, Inc, contributed to the study design; and Stephen Wright, MA, MD, FRCP,
GW Research, Ltd, contributed to the study design. Medical writing support was provided
to the authors by Keira Kim, BS, Greenwich Biosciences, Inc, and Lauren Whyte, PhD,
GW Research, Ltd, at the time the work was completed. None of these contributors received
any compensation other than salary as employees.
