Abstract
Summary
The purpose of this summary is to explain the results from two studies that looked at the safety and effects of an investigational drug called ecopipam. “Investigational” means that the drug has not been approved by the United States Food and Drug Administration (FDA) yet. The studies tested if ecopipam could safely improve symptoms and quality of life in children and adolescents with Tourette syndrome.
Please note that ecopipam is not currently approved to treat Tourette syndrome.
Footnotes
Acknowledgements
Editorial and medical writing assistance were provided under the direction of the authors by Mary Beth Moncrief, PhD, and Rachel Haake, PhD, Synchrony Medical Communications, LLC, West Chester, PA, USA. Funding for this assistance was provided by Emalex Biosciences, Inc., Chicago, IL USA.
Ethics approval and consent to participate
The studies were conducted in accordance with the International Council for Harmonisation Good Clinical Practice and the ethical principles of the Declaration of Helsinki. The study protocols (Pro00033028 [randomized controlled trial]; Pro00037901 [open-label extension study]), including informed consent and assent forms, were approved by a central institutional review board (Advarra, Columbia, MD) and/or the institutional review board or independent ethics committee at each study center. For patients younger than 18 years of age, parents or legal guardians provided written informed consent and patients provided written assent prior to the initiation of any study procedures. Patients who were 18 years old independently provided written informed consent.
Author contributions
Conceptualization, methodology, writing – original draft, writing – review and editing: DL Gilbert and GB Karkanias. Conceptualization, methodology, writing – review and editing: DJB Kim, MM Miller, SD Atkinson, FE Munschauer, SP Wanaski, and TM Cunniff
Funding
Funding for editorial and medical writing assistance was provided by Emalex Biosciences, Inc., Chicago, IL, USA.
Competing interests
DL Gilbert reports being a clinical trial site investigator for Emalex Biosciences, Inc., Neurocrine Biosciences, PTC Therapeutics, and Quince Therapeutics. He has served as a consultant to Emalex Biosciences, Inc., Noema Pharma, PTC Therapeutics, and Vima Therapeutics. He has received book royalties from Wolters Kluwer and Elsevier. DJB Kim, MM Miller, SD Atkinson, GB Karkanias, and FE Munschauer are employees of and have personal equity interest in Emalex Biosciences, Inc. SP Wanaski and TM Cunniff are employees of Paragon Biosciences, which has controlling equity interest in Emalex Biosciences, Inc., and have personal equity interest in Emalex Biosciences, Inc.
Availability of data and material
The data that support the findings of these two studies are available upon reasonable written request.