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Article History

Received: 17 December 2024

Accepted: 10 May 2025

First Online: 11 June 2025

Declarations

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: The trial was conducted in accordance with the revised Declaration of Helsinki (2013), International Conference on Harmonization E6/Good Clinical Practice Guidelines for the conduct of Clinical Trial Investigations (1996) and in compliance with the requirements of the clinical trial regulatory guidelines and regulations. All sites were activated following institutional review board approval.

: JW has received research support from Takeda, Janssen, Chiesi, MustangBio, ADMA Biologics, Octapharma, X4-Pharmaceuticals, Novartis, and Regeneron, Bristol Myers Squibb; has served as a consultant or in advisory boards for Takeda, X4- Pharmaceuticals, CSL-Behring, Grifols, ADMA Biologics, Enzyvant, Regeneron, Pharming, Takeda, and UptoDate. DJ has been a speaker for AstraZeneca and GSK. RA, MA, and MCS are employees of ADMA Biologics. WD serves as a consultant for ADMA Biologics. JD has received research support from Horizon Therapeutics (now Amgen), Prometic, and CSL-Behring; has served as a consultant, speaker, or in advisory boards for Horizon Therapeutics (now Amgen), CSL-Behring, Pharming, ADMA Biologics, Prime Medicine, Rocket Pharma, and Grifols; and was previously an employee and shareholder of Bluebird Bio. Other authors had no conflicts of interest to disclose.

: Written informed consent was obtained from participants’ parent/legal guardian indicating that they understood the study purpose and procedures and were willing to participate.