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Authors
Schiffmann, Raphael https://orcid.org/0000-0003-2437-0252
Mengel, Eugen https://orcid.org/0000-0001-8967-2951
Wallace, Mary
Rochmann, Camille
Turnbull, James
Krupnick, Robert
Gwaltney, Chad
Pulikottil-Jacob, Ruth
Batsu, Isabela
Zheng, Riliang
Hamed, Alaa
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Clinical Trials BETA
Clinical trials referenced in this document:
nct02843035 at ClinicalTrials.govDocuments that mention this clinical trial
Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP)
https://doi.org/10.1007/s12325-024-02881-2
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Funding
Funding for this research was provided by:
Sanofi
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Article History
Received: 15 January 2024
Accepted: 22 April 2024
First Online: 27 May 2024
Declarations
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: Raphael Schiffmann is the Lead Principal Investigator on the venglustat LEAP trial and receives research support and honoraria from Sanofi, Chiesi Pharmaceuticals, and Protalix Biotherapeutics. Eugen Mengel is a Principal Investigator on the venglustat LEAP trial and reports receiving fees and consultant honoraria from Actelion, Alexion, Avrobio, Cyclo Therapeutics, Freeline Therapeutics, Idorsia, Orphazyme A/S, Sanofi, and Takeda. Mary Wallace was the Clinical Study Coordinator on the venglustat LEAP trial. Ruth Pulikottil-Jacob, Camille Rochmann, Isabela Batsu, Riliang Zheng, and Alaa Hamed are employees of Sanofi; Isabela Batsu and Riliang Zheng hold shares and/or stock options in this company. James Turnbull and Robert Krupnick are employees of IQVIA, Inc., which received fees for this study. Chad Gwaltney is an employee of Gwaltney Consulting and was a paid consultant of Sanofi for this study; however, this author did not receive any payment related to authoring this manuscript.
: This standalone qualitative study was not included in the LEAP trial protocol. The study was approved by a third-party institutional review board (Advarra, Columbia, MD) and was conducted in accordance with the Helsinki Declaration of 1964 and its later amendments. Patients and caregivers provided informed consent to participate online or on paper after being approached by the research coordinator from a participating clinical trial site in the US. Patients and caregivers were also asked for verbal confirmation of their consent before the start of their interviews.
