Document is current

Article History

Accepted: 10 September 2023

First Online: 7 October 2023

Declarations

:

: This trial was sponsored by Ipsen.

: RM: Employee of Ipsen; LD: Pharmacokinetic Consultant for Ipsen; JO: Employee of Ipsen; K-HLQS: Coordinator of Ipsen FOP-program and MO-trial.

: The original protocol (version 2.0, dated 14 December 2018; approved 20 December 2018), protocol amendment 1 (dated 7 January 2019; approved 17 January 2019), informed consent forms and updates for this trial were approved by the Institutional Review Board in accordance with pertinent country-specific regulatory requirements, and carried out in compliance with the Declaration of Helsinki and International Council for Harmonisation Good Clinical Practice Guidelines.

: All participants provided written and signed informed consent before engaging in any trial procedure.

: Not applicable.

: Qualified researchers may request access to patient-level study data that underlie the results reported in this publication. Additional relevant study documents, including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan and dataset specifications may also be made available. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of study participants. Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later. Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (). Any requests should be submitted to for assessment by an independent scientific review board.

: Not applicable.

: The authors thank Emma Lockyer, MChem, Jessica A. Buttress, PhD, and Oliver Palmer, BSc (Hons), of Costello Medical, UK, for providing medical writing and editorial support, which was sponsored by Ipsen in accordance with Good Publication Practice guidelines.

: Substantial contributions to study conception and design: RM, LD, JO, K-HLQS; substantial contributions to analysis and interpretation of the data: RM, LD, JO, K-HLQS; drafting the article or revising it critically for important intellectual content: RM, LD, JO, K-HLQS; final approval of the version of the article to be published: RM, LD, JO, K-HLQS.