van Tilburg, Cornelis M.
Kilburn, Lindsay B.
Perreault, Sébastien
Schmidt, Rene
Azizi, Amedeo A.
Cruz-Martínez, Ofelia
Zápotocký, Michal
Scheinemann, Katrin
Meeteren, Antoinette Y. N. Schouten-van
Sehested, Astrid
Opocher, Enrico
Driever, Pablo Hernáiz
Avula, Shivaram
Ziegler, David S.
Capper, David
Koch, Arend
Sahm, Felix
Qiu, Jiaheng
Tsao, Li-Pen
Blackman, Samuel C.
Manley, Peter
Milde, Till
Witt, Ruth
Jones, David T. W.
Hargrave, Darren
Witt, Olaf
Article History
Received: 31 August 2023
Accepted: 2 January 2024
First Online: 30 January 2024
Declarations
:
: The study was conducted in compliance with ICH Good Clinical Practice guidelines and ethical principles described in the Declaration of Helsinki. The study protocol and all amendments were reviewed by the Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for each participating study center. Study centers as of Sept. 6, 2023 include: Perth Childrens Hospital (Nedlands, Australia), Sydney Children’s Hospital (Randwick, Australia), The Royal Children’s Hospital – Children’s Cancer Centre (Parkville, Australia), Women’s and Children’s Health Network (North Adelaide, Australia), Medizinische Universität Wien (Innsbruck, Austria), Cliniques Universitaires Saint-Luc (Brussels, Belgium), Universitair Ziekenhuis Gent (Ghent, Belgium), Centre Hospitalier de l’Université Laval et Centre Mère-Enfant Soleil (Québec, Canada), CHU Sainte-Justine (Québec, Canada), The Montreal Children’s Hospital (Québec, Canada), Fakultní nemocnice Brno—Dětská, Nemocnice (Brno, Czechia), Motol University Hospital (Prague, Czechia), Rigshospitalet (Copenhagen, Denmark), Tampereen Yliopistollinen Sairaala (Tampere, Finland), Hôpital de le Timone (Marseille, France), Institut Curie (Paris, France), Charite Universitätsmedizin Berlin (Berlin, Germany), Evangelische Klinikum Bethel (EvKB) (Bielefeld, Germany), Universitätsklinikum Hamburg-Eppendorf (Hamburg, Germany), Universitätsklinikum Heidelberg (Heidelberg, Germany), Universitätsklinikum Erlangen (Ulm, Germany), Aghia Sofia General Children’s Hospital (Athens, Greece), Athens General Children’s Hospital (Athens, Greece), Semmelweis Egyetem Tűzoltó utcai ll. Sz. Gyermekgyógyászati Kliniká (Budapest, Hungary), Prinses Máxima Centrum Kinderoncologie (Utrecht, Netherlands), Starship Paediatric Blood & Cancer Center (Grafton, New Zealand), Seoul National University Hospital (Seoul, South Korea), Severance Hospital, (Seoul, South Korea), SingHealth Group—KK Women’s and Children’s Hospital (Singapore), Univerzitetni Klinini Center Ljubljana (Ljubljana, Slovenia), Hospital Universitari Vall d’Hebrón (Barcelona, Spain), Hospital Sant Joan de Déu Barcelona (Barcelona, Spain), Hospital Infantil Universitario Niño Jesús (Madrid, Spain), Hospital Universitario Virgen del Rocío (Sevilla, Spain), Hospital Universitari I Politècnic La Fe (Valencia, Spain), Astrid Lindgrens Barnsjukhus Solna (Stockholm, Sweden), Centre Hospitalier Universitaire Vaudois Lausanne (Lausanne, Switzerland), Universitaets—Kinderspital Zürich (Zürich, Switzerland), Ann & Robert H. Lurie Childrens Hospital of Chicago, (Illinois, United States), Arnold Palmer Hospital for Children (Florida, United States), Children’s Hospital Colorado (Colorado, United States), Children’s Hospital Los Angeles (California, United States), Childrens National Medical Center (The District of Columbia, United States), Dana-Farber Cancer Institute (Massachusetts, United States), Duke Cancer Institute (North Carolina, United States), New York University Langone Health (New York, United States), Phoenix Children’s Hospital (Arizona, United States), Riley Hospital for Children at Indiana University Health (Indiana, United States), St. Louis Childrens Hospital (Missouri, United States), Texas Children’s Hospital (Texas, United States), University of Michigan – C.S. Mott Children’s Hospital (Michigan, United States), University of Rochester (New York, United States), and University of Texas Southwestern Medical Center (Texas, United States). Written, informed consent/pediatric assent will be obtained by the investigator and designated site staff and withdrawal of consent/assent will be available at all times during the study.
: Not applicable.
: The authors wish to declare the following competing interests: C.M.vT has participated in advisory boards for Novartis, Bayer, and Alexion. L.B.K has received consulting fees from Blueprint Medicine and contracted research from Novartis, Regeneron Pharmaceuticals, Day One Biopharmaceuticals, Spring Works Therapeutics, Bristol Myers Squibb, and SonALAsense and also is a stock shareholder at Onconova Therapeutics. S.P has participated in advisory boards for Bayer, Alexion, and Esai and received research support from Novartis, Bayer, and Roche. A.A.A has participated in an advisory board for Alexion and received travel and scientific grants from Alexion. M.Z has participated in an advisory board for AstraZeneca. K.S has participated in advisory boards for Alexion, Servier, Jazz Pharmaceuticals, Novartis, Roche, Bayer, and NovoNordisk. A.S has participated in advisory boards for Alexion, AstraZeneca Rare Disease, and Day One Biopharmaceuticals. E.O has participated in an advisory board for Alexion/AstraZeneca Rare Disease. P.HD has participated in an advisory board and consultancy for Alexion. D.S.Z has received consulting fees from Bayer, AstraZeneca, Accendatech, Novartis, Day One Biopharmaceuticals, FivePhusion, Amgen, Alexion, and Norgine and also received research support from Accendatech. D.C is a co-founder and shareholder for Heidelberg Epignostix GmbH and receives royalties for sale of BRAF V600E specific antibody VE1. F.S has received honoraria from Bayer and Illumina and is a co-founder and shareholder for Heidelberg Epignostix GmbH. J.Q, L-P.T, S.C.B, and P.M are all employees of Day One Biopharmaceuticals and have received Day One Biopharmaceuticals stock and stock options. T.M has received research grants from Day One Biopharmaceuticals and Biomed Valley Discovery. D.T.W.J is a co-founder and shareholder for Heidelberg Epignostix GmbH. D.H has participated in advisory boards for Alexion/AstraZeneca, Bayer, Bristol Myers Squibb, Celgene, Day One Biopharmaceuticals, Janssen, Novartis, and Roche and also received research grants from Alexion/AstraZeneca and Roche. O.W has participated in advisory boards for Novartis, Janssen, Roche, Bristol Myers Squibb, and AstraZeneca and also received research grants from Day One Biopharmaceuticals, Biomed Valley Discovery, Bristol Myers Squibb, Syndax, and PreComb. The following authors have no competing interests to declare: R.S, O.C, A. Y. N. SvM, S.A, A.K, and R.W.