{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,13]],"date-time":"2026-06-13T15:32:08Z","timestamp":1781364728006,"version":"3.54.1"},"reference-count":19,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,6,6]],"date-time":"2024-06-06T00:00:00Z","timestamp":1717632000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2024,8]]},"abstract":"Abstract<\/jats:title>Introduction\/Aims<\/jats:title>Expanded access (EA) is a Food and Drug Administration\u2010regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.<\/jats:p><\/jats:sec>Methods<\/jats:title>A central coordination center was established to design and conduct the program. Templated documents and processes were developed to streamline study design, regulatory submissions, and clinical operations across protocols. The program included three protocols and provided access to IPs that were being tested in respective regimens of the HEALEY ALS Platform Trial (verdiperstat, CNM\u2010Au8, and pridopidine). Clinical and safety data were collected in all EA protocols (EAPs). The program cohorts comprised participants who were not eligible for the platform trial, including participants at advanced stages of disease progression and with long disease duration.<\/jats:p><\/jats:sec>Results<\/jats:title>A total of 85 participants were screened across the 3 EAPs from July 2021 to September 2022. The screen failure rate was 3.5%. Enrollment for the regimens of the platform trial was completed as planned and results informed the duration of the corresponding EAP. The verdiperstat EAP was concluded in December 2022. Mean duration of participation in the verdiperstat EAP was 5.8\u2009\u00b1\u20094.1\u2009months. The CNM\u2010Au8 and pridopidine EAPs are ongoing.<\/jats:p><\/jats:sec>Discussion<\/jats:title>Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28169","type":"journal-article","created":{"date-parts":[[2024,6,6]],"date-time":"2024-06-06T11:06:49Z","timestamp":1717672009000},"page":"232-239","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis"],"prefix":"10.1002","volume":"70","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6964-9842","authenticated-orcid":false,"given":"Dylan V.","family":"Neel","sequence":"first","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7906-5752","authenticated-orcid":false,"given":"Clara","family":"Baselga\u2010Garriga","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Molly","family":"Benson","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mackenzie","family":"Keegan","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Chase","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derek","family":"D'Agostino","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin","family":"Drake","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer Linn","family":"Hagar","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith Gibbons","family":"Hasenoehrl","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Kulesa\u2010Kelley","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Leite","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silpa","family":"Mohapatra","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanna Marie","family":"Portaro","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay M.","family":"Pothier","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander V.","family":"Sherman","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hong","family":"Yu","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"McCaffrey","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doreen","family":"Ho","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Luppino","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4666-7495","authenticated-orcid":false,"given":"Richard","family":"Bedlack","sequence":"additional","affiliation":[{"name":"Department of Neurology Duke University Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daragh","family":"Heitzman","sequence":"additional","affiliation":[{"name":"MDA\/ALS Clinic, Texas Neurology Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Senda","family":"Ajroud\u2010Driss","sequence":"additional","affiliation":[{"name":"Les Turner ALS Center Northwestern University Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Katz","sequence":"additional","affiliation":[{"name":"California Pacific Medical Center Research Institute and Forbes Norris MDA\/ALS Research and Treatment Center San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin","family":"Felice","sequence":"additional","affiliation":[{"name":"Department of Neuromuscular Medicine, Hospital for Special Care New Britain Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles","family":"Whitaker","sequence":"additional","affiliation":[{"name":"Department of Neuromuscular Medicine, Hospital for Special Care New Britain Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shafeeq","family":"Ladha","sequence":"additional","affiliation":[{"name":"Gregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute Phoenix Arizona USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gustavo","family":"Alameda","sequence":"additional","affiliation":[{"name":"Holy Cross Hospital Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eduardo","family":"Locatelli","sequence":"additional","affiliation":[{"name":"Center for Collaborative Research Nova Southeastern University and Dr. Kiran C. Patel College of Allopathic Medicine Fort Lauderdale Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irfan A.","family":"Qureshi","sequence":"additional","affiliation":[{"name":"Biohaven Pharmaceuticals, Inc. New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael T.","family":"Hotchkin","sequence":"additional","affiliation":[{"name":"Clene Nanomedicine, Inc. Salt Lake Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael R.","family":"Hayden","sequence":"additional","affiliation":[{"name":"Naarden Prilenia Therapeutics The Netherlands"},{"name":"Center for Molecular Medicine and Therapeutics, British Columbia Children's Hospital Research Institute University of British Columbia Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7075-1681","authenticated-orcid":false,"given":"Merit E.","family":"Cudkowicz","sequence":"additional","affiliation":[{"name":"Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suma","family":"Babu","sequence":"additional","affiliation":[{"name":"Sean M. 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