Funding

Institut national de la santé et de la recherche médicale (Inserm) is the sponsor of the TREOCAPA trial. The project leading to this application has received funding through the Connect4Children consortium from the Innovative Medicines Initiative 2 Joint Undertaking under Grant Agreement n°777389. This joint undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

Role of the funder/sponsor

The funding organisation had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.

Conflict Of Interest

Faheemah PADAVIA, Jean-Marc TRELUYER, Gilles CAMBONIE, Cyril FLAMANT, Aline RIDEAU, Manon TAUZIN, Juliana PATKAI, Géraldine GASCOIN, Mirka LUMIA, Outi AIKIO, Frantz FOISSAC, Saïk URIEN, Sihem BENABOUD, Gabrielle LUI, Léo FROELICHER BOURNAUD, Yi ZHENG, Ruth KEMPER, Marine TORTIGUE, Alban-Elouen BARUTEAU, Jaana KALLIO, Mikko HALLMAN, Alpha DIALLO, Léa LEVOYER, Jean-Christophe ROZE and Naïm BOUAZZA declare that they have no potential conflicts of interest that might be relevant to the contents of this manuscript.

Ethics approval

The trial was approved by the ethics committee of the Centre Hospitalier La Chartreuse under the approval number SI 20.03.09.40128 for France and by the regional medical research ethics committee of North Ostrobothnia under the approval number 68/06.00.00/20 19 for Finland.

Consent to participate

Written informed consent was obtained from both parents.

Consent for publication

Not applicable.

Data availability statement

The data that support the findings of this study are available from Institut national de la santé et de la recherche médicale (Inserm) but restrictions apply to the availability of these data, which were used under license for the current study, and so are not publicly available. Data are, however, available from the authors upon reasonable request and with the permission of Inserm.

Author Contributions

Concept and design: NB, MH, JMT, JCR. Acquisition of data: GC, CF, AR, MT, JP, GG, ML, OA, YZ, MT, AEB. Analysis and interpretation of data: FP, NB, FF, SU, JCR. Drafting of the manuscript: FP, NB, FF, SU, GL, LFB, JCR. Critical revision of the manuscript for important intellectual content: All authors.