A research steering committee comprising of a haematologist, a paediatrician and a paediatric health researcher will be created to ensure effective running of the study. Publication and presentation Timeline and study status One year from approval. Patient recruitment Fingolimod will begin immediately after
registration on clinicaltrials.gov and will continue for 4 months. The detailed study will be completed within 12 months of final recruitment and will be sent to a leading journal for publication. We have currently completed the recruitment of patients for our project in September 2014. All recruited patients have undergone baseline imaging and pertinent laboratory testing and clinical examinations. The participants have been assigned to their respective arms with some of our earlier recruits near to completing the first 6 months of the study. As mentioned in the protocol, repeat imaging and serum ferritin levels will be obtained at 6 months. We anticipate our 6-month imaging to begin from December 2014 and to continue up to March 2015. On the basis of this timeline, we hope to complete our exit 12-month imaging from June 2015 to September 2015
and conclude our study by September 2015. As mentioned earlier, all our study patients will be reviewed in the clinics regularly and managed as per clinical needs. Dissemination of results Findings of this study will be reported through scientific publications and research conferences. Additionally, the findings will also be disseminated to the participants after project completion through a project summary and conclusion document distributed in an exit meeting and pertinent patient awareness sessions will be planned with the participants after the conclusion of the project. Additional information about our future work and study plans as well as clinical implications and future management regimens will also be discussed. Supplementary Material Author’s manuscript: Click here to view.(11M, pdf) Reviewer comments: Click here to view.(144K, pdf) Footnotes Contributors:
BSH Brefeldin_A is the guarantor of the trial and responsible for the trial conception and design. BSH is also the contact person for all published versions of the protocol and manuscript. BSH, ZF, MZ and SC were involved in the conception of this trial. BSH, AS and FQ were involved in obtaining institutional approval. In addition to these authors, AS, ASh and NA were involved in the development of the protocol. AR was the study statistician. FAT performed patient MRIs. BSH performed patient echocardiography. ASh and NA were responsible for patient recruitment. NA was involved in data collection and maintenance of trial data and patient supervision. AR, NA and AS conducted data analysis. In addition to these authors, BSH also assisted in data interpretation.