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This ongoing open-label extension (OLE; NCT04138277) evaluates long-term safety and efficacy of cipa\u2009+\u2009mig. Outcomes include 6-min walk distance (6MWD), forced vital capacity (FVC), creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, patient-reported outcomes and safety. Data are reported as change from PROPEL baseline to OLE week 52 (104 weeks post-PROPEL baseline). Of 118 patients treated in the OLE, 81 continued cipa\u2009+\u2009mig treatment from PROPEL (cipa\u2009+\u2009mig group; 61 enzyme replacement therapy [ERT] experienced prior to PROPEL; 20 ERT na\u00efve) and 37 switched from alg\u2009+\u2009pbo to cipa\u2009+\u2009mig (switch group; 29 ERT experienced; 8 ERT naive). Mean (standard deviation [SD]) change in %\u00a0predicted 6MWD from baseline to week 104 was\u2009+\u20093.1 (8.1) for cipa\u2009+\u2009mig and\u2009\u2212\u20090.5 (7.8) for the ERT-experienced switch group, and\u2009+\u20098.6 (8.6) for cipa\u2009+\u2009mig and\u2009+\u20098.9 (11.7) for the ERT-na\u00efve switch group. Mean (SD) change in % predicted FVC was\u2009\u2212\u20090.6 (7.5) for cipa\u2009+\u2009mig and\u2009\u2212\u20093.8 (6.2) for the ERT-experienced switch group, and\u2009\u2212\u20094.8 (6.5) and\u2009\u2212\u20093.1 (6.7), respectively, in ERT-na\u00efve patients. CK and Hex4 levels improved in both treatment groups by week 104 with cipa\u2009+\u2009mig treatment. Three patients discontinued the OLE due to infusion-associated reactions. No new safety signals were identified. Cipa\u2009+\u2009mig treatment up to 104 weeks was associated with overall maintained improvements (6MWD, biomarkers) or stabilization (FVC) from baseline with continued durability, and was well\u00a0tolerated, supporting long-term benefits for patients with LOPD.<\/jats:p><jats:p><jats:bold>Trial registration number<\/jats:bold>: NCT04138277; trial start date: December 18, 2019.<\/jats:p>","DOI":"10.1007\/s00415-024-12236-0","type":"journal-article","created":{"date-parts":[[2024,2,28]],"date-time":"2024-02-28T19:02:37Z","timestamp":1709146957000},"page":"2810-2823","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":34,"title":["104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study 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He has participated as a scientific advisor for Amicus Therapeutics Inc., Argenx, Astellas, Bayer, Maze, Pepgen, Sanofi, Spark, and Taysha. He declares no stocks or shares. Drago Bratkovic declares no competing interests. Barry J. Byrne has participated as a consultant\/advisory board member for Pfizer, Amicus Therapeutics, Inc., and Sanofi. He also owns stocks in Lacerta Therapeutics. Kristl G. Claeys received research funding from Alnylam, Biogen, Pfizer, Roche, Sanofi-Genzyme; received advisory board member honoraria from Alexion, Alnylam, Amicus, ArgenX, Biogen, Ipsen, Janssen Pharmaceutics, Lupin, Pfizer, Roche, Sanofi-Genzyme and UCB and is Chairholder of the Emil von Behring Chair for Neuromuscular and Neurodegenerative Disorders by CSL Behring. Jordi Diaz-Manera has received consulting fees\/honoraria from Sarepta, Sanofi and Audentes and grant support from Sanofi, Spark and Boehringer Ingelheim. He has received payment for speaking from Sanofi, Sarepta and Lupin. Priya S. Kishnani has received research\/grant support from Sanofi Genzyme and Amicus Therapeutics Inc. and has received consulting fees and honoraria from Sanofi Genzyme, Amicus Therapeutics, Inc., Maze Therapeutics, Bayer and Asklepios Biopharmaceutical, Inc. (AskBio). She is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Pompe Disease Advisory Board for Amicus Therapeutics Inc., and Advisory Board for Baebies. Priya S. Kishnani has equity with Maze Therapeutics and has held equity in Asklepios Biopharmaceuticals and may receive milestone payments related to that equity in the future. Pascal Laforet is a consultant\/advisory board member for Amicus Therapeutics Inc., Sanofi Genzyme and Spark Therapeutics. He has received travel expenses from Sanofi Genzyme and Spark Therapeutics. Mark Roberts has received honorarium for educational symposia from Sanofi Genzyme and Amicus and for participation on advisory boards for Sanofi, and Amicus. Antonio Toscano received honorarium for educational talks from Sanofi Genzyme and Amicus and for participation on advisory boards for Sanofi, Amicus, Aro and Spark. He is a member of the European Reference Network for Neuromuscular Disorders (EU-NMD),\u00a0Project ID 739543. Ans T. van der Ploeg is an advisory board member of Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics. She has provided consultancies for Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics and has contracted research for Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics. All collaborations are done under an agreement between Erasmus MC and these industries. Tahseen Mozaffar has participated in an advisory capacity for Abbvie, Alexion, Amicus Therapeutics, Inc., Annji, Argenx, Arvinas, Audentes, Cabaletta, Maze Therapeutics, Momenta, Ra Pharmaceuticals, Sanofi Genzyme, Sarepta, Spark Therapeutics, and UCB, and has participated in the speaker\u2019s bureau for Sanofi Genzyme. He is a member of the medical advisory board for the Myositis Association, Neuromuscular Disease Foundation, Myasthenia Gravis Foundation of California and Myasthenia Gravis Foundation of America. He has received research funding from the Myositis Association, the Muscular Dystrophy Association, the NIH and from the following sponsors: Alexion, Amicus Therapeutics, Inc., Annji, Argenx, Audentes, Bristol-Myers Squib, Cabaletta, Cartesian Therapeutics, Grifols, Momenta, Ra Pharmaceuticals, Sanofi Genzyme, Spark Therapeutics, UCB, and Valerion. He is a member of the Data safety monitoring board for Acceleron, Applied Therapeutics, Sarepta, and the NIH. Jeff Castelli, Mitchell Goldman, Fred Holdbrook, Sheela Sitaraman Das, and Yasmine Wasfi are employees of, and hold stocks and shares in, Amicus Therapeutics, Inc.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"This study was conducted in accordance with ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments, and in compliance with the United States (US) Food and Drug Administration (FDA) regulations in 21 Code of Federal Regulations 56, European Union (EU) Clinical Trials Regulation 536\/2014, and the International Council for Harmonisation Good Clinical Practice (GCP) guidelines. For each study site, the clinical study protocol (and any amendments) and informed consent form were reviewed and approved by the appropriate Independent Ethics Committee (IEC)\/Institutional Review Board (IRB).","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"All patients provided signed informed consent before any study-related procedures were performed. In Japan, the patient\u2019s parental guardian (or legal representative) also had to sign the informed consent form for patients under 20\u00a0years of age.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent"}}]}}