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We report data from the Phase I\/II ATB200-02 study for up to 48\u00a0months of treatment. Four adult cohorts, including one non-ambulatory ERT-experienced (<jats:italic>n<\/jats:italic>\u2009=\u20096) and three ambulatory cohorts, (two enzyme replacement therapy [ERT]-experienced cohorts [2\u20136 years (<jats:italic>n<\/jats:italic>\u2009=\u200911) and\u2009\u2265\u20097 years (<jats:italic>n<\/jats:italic>\u2009=\u20096)]), one ERT-na\u00efve cohort (<jats:italic>n<\/jats:italic>\u2009=\u20096), received 20 mg\/kg intravenous-infused cipa plus 260 mg oral mig biweekly. Change from baseline (CFBL) for multiple efficacy endpoints at 12, 24, 36, and 48 months, pharmacodynamics, pharmacokinetics, safety, and immunogenicity data were assessed. Six-minute walking distance (% predicted) improved at 12, 24, 36, and 48\u00a0months: pooled ambulatory ERT-experienced cohorts, mean(\u00b1\u2009standard deviation [SD]) CFBL: 6.1(\u00b1\u20097.84), <jats:italic>n<\/jats:italic>\u2009=\u200916; 5.4(\u00b1\u200910.56), <jats:italic>n<\/jats:italic>\u2009=\u200913; 3.4(\u00b1\u200914.66), <jats:italic>n<\/jats:italic>\u2009=\u200912; 5.9(\u00b1\u200917.36), <jats:italic>n<\/jats:italic>\u2009=\u20099, respectively; ERT-na\u00efve cohort: 10.7(\u00b1\u20093.93), <jats:italic>n<\/jats:italic>\u2009=\u20096; 11.0(\u00b1\u20095.06), <jats:italic>n<\/jats:italic>\u2009=\u20096; 9.0(\u00b1\u20097.98), <jats:italic>n<\/jats:italic>\u2009=\u20095; 11.7(\u00b1\u20097.69), <jats:italic>n<\/jats:italic>\u2009=\u20094, respectively. Percent\u00a0predicted forced vital capacity was generally stable in ERT-experienced cohorts, mean(\u00b1\u2009SD) CFBL\u2009\u2212\u20091.2(\u00b1\u20095.95), <jats:italic>n<\/jats:italic>\u2009=\u200916; 1.0(\u00b1\u20097.96), <jats:italic>n<\/jats:italic>\u2009=\u200913;\u2009\u2212\u20090.3(\u00b1\u20096.68), <jats:italic>n<\/jats:italic>\u2009=\u200910; 1.0(\u00b1\u20096.42), <jats:italic>n<\/jats:italic>\u2009=\u20096, respectively, and improved in the ERT-na\u00efve cohort: 3.2(\u00b1\u20098.42), <jats:italic>n<\/jats:italic>\u2009=\u20096; 4.7(\u00b1\u20095.09), <jats:italic>n<\/jats:italic>\u2009=\u20096; 6.2(\u00b1\u20093.35), <jats:italic>n<\/jats:italic>\u2009=\u20095; 8.3(\u00b1\u20094.50), <jats:italic>n<\/jats:italic>\u2009=\u20094, respectively. Over 48\u00a0months, CK and Hex4 biomarkers improved in ambulatory cohorts. Overall, cipa\u2009+\u2009mig was well tolerated with a safety profile like alglucosidase alfa. ATB200-02 results show the potential benefits of cipa\u2009+\u2009mig as a long-term treatment option for Pompe disease. <jats:italic>Trial registration number<\/jats:italic>: NCT02675465 January 26, 2016.<\/jats:p>","DOI":"10.1007\/s00415-023-12096-0","type":"journal-article","created":{"date-parts":[[2023,12,6]],"date-time":"2023-12-06T18:03:15Z","timestamp":1701885795000},"page":"1787-1801","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":25,"title":["Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I\/II study (ATB200-02)"],"prefix":"10.1007","volume":"271","clinical-trial-number":[{"clinical-trial-number":"nct02675465","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7302-1756","authenticated-orcid":false,"given":"Barry J.","family":"Byrne","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya S.","family":"Kishnani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Drago","family":"Bratkovic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula R.","family":"Clemens","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ozlem","family":"Goker-Alpan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xue","family":"Ming","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Roberts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthias","family":"Vorgerd","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kumaraswamy","family":"Sivakumar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ans T.","family":"van der Ploeg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitchell","family":"Goldman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacquelyn","family":"Wright","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fred","family":"Holdbrook","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vipul","family":"Jain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elfrida R.","family":"Benjamin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franklin","family":"Johnson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheela Sitaraman","family":"Das","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasmine","family":"Wasfi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,12,6]]},"reference":[{"key":"12096_CR1","first-page":"1","volume":"7","author":"J Cabello","year":"2017","unstructured":"Cabello J, Marsden D (2017) Pompe disease: clinical perspectives. 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Byrne has participated as a consultant\/advisory board member for Pfizer, Amicus Therapeutics, Inc., and Sanofi. He also owns stocks in Lacerta Therapeutics. Benedikt Schoser has received unrestricted research grants from Amicus Therapeutics, Inc., Astellas, Roche, Marigold Foundation, and the AMDA Foundation and speaker\u2019s honoraria from Amicus Therapeutics, Inc., Alexion, Kedrion, and Sanofi. He has participated as a scientific advisor for Amicus Therapeutics, Inc., Argenx, Astellas, Maze, Pepgen, Sanofi, Spark, and Taysha. He declares no stocks or shares. 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, the Pompe Disease Advisory Board for Amicus Therapeutics, Inc., and the 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. Paula R. Clemens has received grant support from Amicus Therapeutics, Inc., Sanofi Genzyme, Spark, ReveraGen, NS Pharma, MDA, NIH, FDA, and Foundation to Eradicate Duchenne. She received an educational honorarium to speak on Pompe disease by Catalyst Medical Education. She was a consultant for Epirium Bio Inc. Ozlem Goker-Alpan has received consulting fees from Sanofi Genzyme, Amicus Therapeutics, Inc., BioMarin, Sanofi, Shire HGT, and Takeda. She has received grants from Sanofi-Genzyme, Amicus Therapeutics, Inc., Freeline, Genentech, Protalix, Sangamo, Sanofi, and Takeda. Mark Roberts has received fees for advisory boards and speaker honoraria from Sanofi, BioMarin, and Amicus Therapeutics, Inc. Matthias Vorgerd has received speaker\u2019s honoraria including travel costs from Hormosan-Pharma, Sanofi Genzyme, and Amicus Therapeutics, Inc. 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., Argenx, Audentes, Maze Therapeutics, Modis, Momenta, Ra Pharmaceuticals, Sanofi Genzyme, Sarepta, Spark Therapeutics, UCB, and Ultragenyx, 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., Argenx, Audentes, Bristol-Myers Squib, 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, Avexis, Sarepta, and the NIH. Mitchell Goldman, Jacquelyn Wright, Fred Holdbrook, Vipul Jain, Elfrida Benjamin, Franklin Johnson, Sheela Sitaraman Das, and Yasmine Wasfi are employees of Amicus Therapeutics, Inc. and hold stocks and shares in Amicus Therapeutics, Inc. Drago Bratkovic, Xue Ming, and Kumaraswamy Sivakumar declare no competing interests.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"This study was designed and monitored in accordance with the ethical principles of Good Clinical Practice guidelines and the Declaration of Helsinki. The clinical study protocol was reviewed and approved by the appropriate independent ethics committee or institutional review board at each study site.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}},{"value":"All participants provided written informed consent before participating in the study.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}}]}}