{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,11]],"date-time":"2026-02-11T01:27:01Z","timestamp":1770773221331,"version":"3.50.0"},"reference-count":31,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2025,8,26]],"date-time":"2025-08-26T00:00:00Z","timestamp":1756166400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["UF1NS131791"],"award-info":[{"award-number":["UF1NS131791"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2025,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)\u2010funded EAP for amyotrophic lateral sclerosis (ALS).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Participants received trehalose, a drug studied in a parallel RCT, for up to 24\u2009weeks; clinical and biomarker data were collected throughout the study.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was \u22120.005, SE\u2009=\u20090.0078; 95% CI (\u22120.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale\u2014Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were \u22125.6 (0.67); 95% CI (\u22127.0, \u22124.3) and \u22124.53 (4.308); 95% CI (\u221213.55, 4.48)], respectively. No unexpected treatment\u2010related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>This EAP establishes a framework for implementing multi\u2010center EAPs that complement data collected from RCTs. Additional NIH\u2010funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study.<\/jats:p>\n                    <jats:p>\n                      <jats:bold>Trial Registration:<\/jats:bold>\n                      ClinicalTrials.gov: NCT05597436\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70011","type":"journal-article","created":{"date-parts":[[2025,8,26]],"date-time":"2025-08-26T14:45:09Z","timestamp":1756219509000},"page":"1108-1116","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis"],"prefix":"10.1002","volume":"72","author":[{"given":"Brooke","family":"Krivickas","sequence":"first","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-9427-1452","authenticated-orcid":false,"given":"Erica","family":"Scirocco","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9233-235X","authenticated-orcid":false,"given":"Elisa","family":"Giacomelli","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Saloni","family":"Sharma","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Molly","family":"Benson","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1305-8124","authenticated-orcid":false,"given":"Mackenzie","family":"Keegan","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Kulesa\u2010Kelley","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6293-806X","authenticated-orcid":false,"given":"Lori B.","family":"Chibnik","sequence":"additional","affiliation":[{"name":"Sean M. 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Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marianne","family":"Chase","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristin","family":"Drake","sequence":"additional","affiliation":[{"name":"Sean M. Healey &amp; AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Silpa","family":"Mohapatra","sequence":"additional","affiliation":[{"name":"Sean M. 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