Funding for this research was provided by:
Université de Lausanne
Received: 8 December 2020
Accepted: 10 March 2021
First Online: 7 April 2021
: F Fakhouri has received fees for expertise, consultancy, and scientific symposia from Alexion Pharmaceuticals, Inc. M Scully has received speaker fees and is on an advisory board for Alexion Pharmaceuticals, Inc. G Ardissino has received speaker fees and is on an advisory board for Alexion Pharmaceuticals, Inc. I Al-Dakkak is an employee of, and may own stocks/options in Alexion Pharmaceuticals, Inc. B Miller was an employee of Alexion Pharmaceuticals, Inc. at the time of this study. E Rondeau has received fees for expertise, consultancy, and scientific symposia from Alexion Pharmaceuticals, Inc.
: A written informed consent was obtained from each patient prior to participation in the study. The Sponsor or its designee could provide an informed consent template to the sites, if required. If the site made any institution-specific modifications, the Sponsor or its designee could review the consent prior to IRB/EC submission. The Investigator or the Sponsor would then submit the approved, revised consent to the appropriate IRB/EC for review and approval prior to the start of the study. If the consent form was revised during the course of the study, all active participating patients to whom the revision may have had an impact must have signed the revised form. Before recruitment and enrollment, each patient was given a full explanation of the study and was allowed time to read the approved informed consent form. Once the Investigator was assured that the individual understood the implications of participating in the study, the patient was asked to give consent to participate in the study by signing the informed consent form. The Investigator provided a copy of the signed informed consent to the patient. The original form was maintained in the study files at the site.
: The sponsors of this study (Alexion Pharmaceuticals, Inc.) have provided consent for publication.
: This was a multicenter study comprising many different sites of enrollment. Federal, provincial, and local regulations and International Conference on Harmonization (ICH) guidelines, if relevant, required that approval was obtained from an Ethics Committee (EC)/Independent review Board (IRB) prior to participation of patients in research studies. Where required and prior to the study onset, the EC/IRB must have approved the protocol, Informed Consent, advertisements to be used for patient recruitment, and any other written information regarding this study to be provided to the patient or the patient’s parents/legal guardian. The sites maintained and made available for review by the Sponsor or its designee documentation of all EC/IRB approvals and of the EC/IRB compliance with ICH Guidance E6: Good Clinical Practice, if relevant. All EC/IRB approvals were signed by the EC/IRB Chairman or designee and identified the EC/IRB name and address, the clinical protocol by title and/or protocol number, and the date approval and/or favorable opinion was granted. The Investigator conducted all aspects of this study in accordance with all national, provincial, and local laws of the pertinent regulatory authorities.