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Article History

Received: 6 August 2025

Accepted: 31 January 2026

First Online: 9 March 2026

Declarations

:

: Access to participant-level data presented in this article and supporting clinical documents with external researchers who provide methodologically sound scientific proposals will be available upon reasonable request for products or indications that have been approved by regulators in the relevant markets and subject to review. Such requests can be made to: Moderna Inc., 325 Binney St., Cambridge, MA 02142 USA or at data_sharing@modernatx.com. A materials transfer and/or data access agreement with the sponsor will be required for accessing shared data. All other relevant data are presented in the paper. The protocol is available online at ClinicalTrials.gov: NCT03484767; registered on 2 April 2018.

: This study abided by all applicable laws and regulations including but not limited to Good Clinical Practices (GCP), Good Pharmacoepidemiology Practices (GPP), Good Pharmacovigilance Practices (GVP), the European Network of Centers for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Guide on Methodological Standards in Pharmacoepidemiology, the ethical principles that have their origins in the Declaration of Helsinki, and applicable privacy laws. Consistent with local regulations and prior to enrollment of participants at a given site, the study protocol was submitted together with its associated documents (eg, ICF) to the responsible Institutional Review Board/Independent Ethics Committee (IRB/IEC) for its review. Participant enrollment did not start at any site before the site obtained written confirmation of a favorable opinion/approval from the relevant central or local IRB/IEC and an approved informed consent/assent form. (IRB: 00104172).

: Not applicable.

: BCS reports clinical trial sponsorship (paid to institution) and advisory board participation for Moderna, Inc. GTB: nothing to disclose HJV: reports research funding and advisory board participation for Moderna, Inc. HL: reports research funding from Moderna, Inc. JLM: nothing to disclose MS reports consulting fees from IECure, LogicBio, and Ultragenyx; honoraria from Immedica; and clinical trial sponsorship from Arcturus and Moderna, Inc BC: nothing to disclose. JDH: has been a consultant for Moderna, Inc. JV: Received consulting fees and research support from Moderna, Inc. CL: nothing to disclose. DK: DK has served as a consultant for Sangamo Therapeutics, Moderna, Genzyme Sanofi, Amicus, and Vertex; has received grant support from Viking Therapeutics, Genzyme Sanofi, Roivant Rare Diseases, and Amicus; and has held equity in Asklepios Biopharmaceutical (AskBio), which is developing gene therapy for Pompe disease. BKB: has received consulting fees and research support from Moderna, Inc. for service on a DSMB. SG: has conducted clinical trials and received consultancy fees and/or honoraria from Ultragenyx, Origin, Zevra, Glycomine and Moderna, Inc. GAD: nothing to disclose. CF: Received grant support from Moderna, Inc.TM: has conducted clinical trials for LogicBio, Sanofi/Genzyme, Mallinckrodt, and previously served as an employee of Novartis and of Biomarin Pharmaceutical, Inc. JL: employee of Moderna, Inc at the time of the study and holds stock in Moderna, Inc. HA: employee of Moderna, Inc at the time of the study and holds stock in Moderna, Inc. ML: employee of and holds stock in Moderna, Inc. SP: employee of and holds stock in Moderna, Inc. VS: employee of and holds stock in Moderna, Inc.