Document is current

Article History

Received: 6 March 2024

Accepted: 29 October 2024

First Online: 11 February 2025

Declarations

:

: This study was conducted at three study sites in Thailand, Türkiye, and the US. The study protocol was approved by the local institutional review boards and/or ethics committees prior to study initiation. All patients or their legally authorized representatives provided written informed consent prior to study enrollment. This study was conducted in accordance with the principles of the Declaration of Helsinki and Council for International Organizations of Medical Sciences International Ethical Guidelines and was consistent with Good Clinical Practices of the International Conference on Harmonization and applicable regulatory requirements. Monitoring and site supervision were performed with oversight by the sponsor.

: Not applicable.

: AO is a consultant and steering committee member for Regeneron, Pharmaceuticals, Inc.; received sample analysis support for a prior collaborative study () from Regeneron Pharmaceuticals, Inc.; and has a pending patent on C5 inhibitor treatment in CHAPLE disease. VC received support to conduct the study and provision of the investigational product from Regeneron Pharmaceuticals, Inc. JJJ, TB, and LP are Regeneron Pharmaceuticals, Inc. employees/stockholders. OAH is an employee/stockholder and has a pending patent on C5 inhibitor treatment in CHAPLE disease. MJL received support for a federally approved CRADA to support the clinical trial and has a pending patent on C5 inhibitor treatment in CHAPLE disease. LLK, SO, AY, PM, MW, and SP are employees of Adelphi Values who received sponsorship through Regeneron Pharmaceuticals, Inc. to conduct the study. DMTB was employed by Adelphi Values at the time this research was conducted though sponsorship by Regeneron Pharmaceuticals, Inc.