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Article History

Received: 22 July 2022

Accepted: 24 October 2023

First Online: 13 November 2023

Declarations

:

: All studies described here were conducted in accordance with the principles that have their origin in the Declaration of Helsinki, inclusive of any subsequent amendment(s), and that are consistent with the International Council for Harmonization (ICH) Good Clinical Practice (GCP), European Union Directive 2001/20/EC, United States Food and Drug Administration (FDA) Code of Federal Regulations, and other applicable local regulatory requirement(s), whichever affords greater patient protection. The original protocol, all protocol amendments, and informed consent/assent forms and updates were reviewed and approved by the corresponding Institutional Review Board (IRB) or Independent Ethics Committee (IEC) at each site in accordance with local regulatory requirements.

: The IRBs/IECs that reviewed and approved the study documents for PVO-1A-001 were: University of Pennsylvania Office of Regulatory Affairs, US; Human Research Protection Program Institutional Review Board, University of California San Francisco, US; Comité de Protection des Personnes Île de France II, Hôpital Necker Enfants Malades, France; Comitato Etico Regionale, IRCCS AOU San Martino IST, Italy; Comite de Etica de Protocolos de Investigacion, Hospital Italiano de Buenos Aires, Argentina; London – Stanmore Research Ethics Committee, Health Research Authority, UK; and Metro South Human Research Ethics Committee, Australia.

: The IRBs/IECs that reviewed and approved the trial documents for PVO-1A-201 were: Ethics Committee Île de France VIII, France; Human Research Protection Program Committee on Human Research, University of California, San Francisco, US; Institutional Review Board, University of Pennsylvania, US; and NRES Committee South East Coast – Brighton & Sussex, UK.

: The IRBs/IECs that reviewed and approved the study documents for PVO-1A-202 were: University of Pennsylvania Institutional Review Board, US; University of California San Francisco Human Research Protection Program Institutional Review Board, US; Mayo Clinic Institutional Review Boards, US; Comité de Protection des Personnes Île de France VIII, Hôpital Ambroise Paré, France; NHS Health Research Authority London – Brighton & Sussex Research Ethics Committee, UK; Comité de Ética de Protocolos de Investigacion, Argentina; and Metro South Human Research Ethics Committee, Australia.

: Prior to participation in the studies, the Investigator and/or delegate fully explained to the patients and/or parents all aspects of the study that were relevant to the decision to participate. Informed consent was sent to the patients and/or parents for reading and reflection, and was documented by means of a written, signed, and dated consent form, prior to study initiation.

: Not applicable.

: These studies were sponsored by Ipsen. Ipsen contributed to the design of the studies, the collection, analysis, and interpretation of data, and the writing of the manuscript. All costs associated with development of this manuscript were funded by Ipsen. In April 2019, Ipsen acquired Clementia Pharmaceuticals.<b>RJP</b>: Research investigator: Clementia/Ipsen, Regeneron; Advisory board: President of the International Clinical Council on FOP; <b>MAM</b>: Research support: Clementia/Ipsen, Regeneron; Non-paid consultant: Biocryst, Blueprint Medicines, Daiichi Sankyo, Incyte, Keros; Advisory board (all voluntary): IFOPA Registry Medical Advisory Board, Incyte, International Clinical Council on FOP; Non-restricted educational fund from Excel and Catalyst sponsored by Ipsen; <b>GB</b>: Advisory board: Clementia/Ipsen, FOP European Consortium, International Clinical Council on FOP; Speaker: Clementia/Ipsen; <b>MAB</b>: Advisory board: Abbvie, Janssen, Pfizer, UCB Pharma, Novartis, Xinthera; Grant support: Abbvie; Research investigator: Abbvie, Clementia/Ipsen, Janssen, Novartis, Pathios, Regeneron; Data Monitoring Safety Committee: Incyte, Ipsen, Regeneron; Speaker: Abbvie, Janssen, Novartis, Pfizer, Regeneron, UCB Pharma; <b>CDC</b>: Research investigator: Clementia/Ipsen; Speaker: Novartis; <b>ECH</b>: Advisory board (all voluntary): Fibrous Dysplasia Foundation, IFOPA Registry medical advisory board, International Clinical Council on FOP; Clinical research support: Clementia/Ipsen, Neurocrine Biosciences Inc., Regeneron; Research investigator: Clementia/Ipsen; <b>RK</b>: Research investigator: Ipsen/Clementia, Kyowa Kirin, Regeneron; Advisory board: IFOPA FOP Registry Medical Advisory Board, International Clinical Council on FOP; <b>K‑HLQS</b>: Coordinator of Ipsen FOP-program and MO-trial; <b>DRG</b>: Chief Medical Officer of Clementia and a shareholder at the time these data were obtained; <b>RM</b>: Employee of Ipsen; <b>ARS</b>: Employee of Ipsen; <b>FSK</b>: Research investigator: Clementia/Ipsen, Regeneron; Advisory Board: IFOPA Medical Advisory Board; Founder and Immediate Past-President of the International Clinical Council on FOP; Chair of the Publications Committee of the International Clinical Council. In April 2019, Ipsen acquired Clementia Pharmaceuticals.