Mekahli, Djalila
Liebau, Max C. http://orcid.org/0000-0003-0494-9080
Cadnapaphornchai, Melissa A. http://orcid.org/0000-0002-0460-2477
Goldstein, Stuart L. http://orcid.org/0000-0003-3711-8785
Greenbaum, Larry A. http://orcid.org/0000-0002-2490-021X
Litwin, Mieczyslaw http://orcid.org/0000-0002-5241-2483
Seeman, Tomas
Schaefer, Franz http://orcid.org/0000-0001-7564-9937
Guay-Woodford, Lisa M. http://orcid.org/0000-0002-6187-3927
Article History
Received: 13 September 2022
Accepted: 30 January 2023
First Online: 13 February 2023
Declarations
:
: The trial is being performed in compliance with International Council for Harmonization Good Clinical Practice guidance, international ethical principles derived from the Declaration of Helsinki and Council for International Organizations of Medical Science guidelines, and applicable local laws and regulations. Each trial site must obtain approval/favorable opinion by an institutional review board or independent ethics committee according to regional requirements. The institutional review boards that have issued approvals to date for Study 204 are: WCG IRB – Puyallup, WA; and Cincinnati Children’s Hospital Institutional Review Board – Cincinnati, OH. The institutional review boards that have issued approvals to date for Study 307 are: WCG IRB – Puyallup, WA; Advarra IRB – Columbia, MD; and Cincinnati Children’s Hospital Institutional Review Board – Cincinnati, OH.Informed consent is obtained from all subjects’ parents or their legal guardians. In Study 307, written informed assent is obtained from all subjects old enough per local laws to provide assent. Age-appropriate assent documents were created and subjects who are able are required to reconsent or assent as appropriate if they matriculate from one age group to another.
: Not applicable.
: DM serves on an advisory board of Otsuka Pharmaceuticals, Sanofi Genzyme and Reata as a representative of the University Hospital of Leuven and has received educational grants from Otsuka Pharmaceuticals paid to the University Hospital of Leuven. All of these activities are outside the submitted work.MCL serves on an advisory board of Otsuka Pharmaceuticals as a representative of the University Hospital of Cologne.MAC serves on a pediatric advisory board of Otsuka Pharmaceuticals Inc.SLG has received grant funding from and served as a consultant for Otsuka.LAG is a consultant for and receives research support from Otsuka Pharmaceuticals.ML reports consulting for Otsuka.TS reports consulting for Otsuka.FS reports consulting for Otsuka.LMGW serves a consultant for and receives honoraria from Otsuka and Natera, Inc.