{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,17]],"date-time":"2026-07-17T00:56:24Z","timestamp":1784249784047,"version":"3.55.0"},"reference-count":15,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"7","license":[{"start":{"date-parts":[[2022,3,3]],"date-time":"2022-03-03T00:00:00Z","timestamp":1646265600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":["Hepatol Commun"],"published-print":{"date-parts":[[2022,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:p>The purpose of this pilot study was to explore the efficacy, safety, and tolerability of vidofludimus calcium (VC) in the treatment of primary sclerosing cholangitis (PSC). This was a single\u2010arm open\u2010label pilot study with a cohort of 18 patients with PSC. Study patients received VC for a period of 6 months. The study was undertaken at two sites, Mayo Clinic, Rochester, MN, and Mayo Clinic, Phoenix, AZ. The primary endpoint of the study was improvement of serum alkaline phosphatase (ALP) at the end of the study. Secondary endpoints included assessment of other liver biomarkers (bilirubin, alanine aminotransferase, and aspartate aminotransferase). Of 18 patients enrolled, 11 completed the 6 months of study treatment. Patients who completed treatment versus those who did not were similar other than a significantly higher direct bilirubin at baseline in the group that completed treatment (mean \u00b1 SD, 0.4 \u00b1 0.3 versus 0.1 \u00b1 0.1, p\u00a0=\u00a00.04). By intent to treat analysis, the primary outcome was met in 16.7% (3\/18) of patients. By per\u2010protocol analysis, including only patients who completed treatment, normalization of ALP occurred in 27.7% (3\/11) at week 24 (95% confidence interval, 6.0% to 61.0%). VC was well tolerated with no drug\u2010related serious adverse events. <jats:italic toggle=\"yes\">Conclusion:<\/jats:italic> This proof of concept study provides support for further exploration of VC in patients with PSC.<\/jats:p>","DOI":"10.1002\/hep4.1926","type":"journal-article","created":{"date-parts":[[2022,3,3]],"date-time":"2022-03-03T11:01:56Z","timestamp":1646305316000},"page":"1589-1597","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":14,"title":["A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis"],"prefix":"10.1097","volume":"6","author":[{"given":"Elizabeth J.","family":"Carey","sequence":"first","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Mayo Clinic,Phoenix,Arizona,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Eaton","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Mayo Clinic,Rochester,Minnesota,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitchell","family":"Clayton","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Mayo Clinic,Rochester,Minnesota,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Gossard","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Mayo Clinic,Rochester,Minnesota,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Iqbal","sequence":"additional","affiliation":[{"name":"Gastroenterology,West Virginia University,Morgantown,West Virginia,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hamid","family":"Ullah","sequence":"additional","affiliation":[{"name":"Gastroenterology,West Virginia University,Morgantown,West Virginia,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nan","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Quantitative Health Research,Mayo Clinic,Phoenix,Arizona,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Butterfield","sequence":"additional","affiliation":[{"name":"Department of Quantitative Health Research,Mayo Clinic,Phoenix,Arizona,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keith\u00a0D.","family":"Lindor","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology,Mayo Clinic,Phoenix,Arizona,USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2022,3,3]]},"reference":[{"key":"hep41926-bib-0001-20241115","doi-asserted-by":"crossref","first-page":"1587","DOI":"10.1016\/S0140-6736(13)60096-3","article-title":"Primary sclerosing 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Pharmacokinet"},{"key":"hep41926-bib-0012-20241115","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1007\/s40268-019-00286-z","article-title":"The selective oral immunomodulator vidofludimus in patients with active rheumatoid arthritis: safety results from the COMPONENT study","volume":"19","year":"2019","journal-title":"Drugs R D"},{"key":"hep41926-bib-0013-20241115","doi-asserted-by":"crossref","first-page":"102129","DOI":"10.1016\/j.msard.2020.102129","article-title":"Vidofludimus calcium, a next generation DHODH inhibitor for the treatment of relapsing\u2010remitting multiple sclerosis","volume":"43","year":"2020","journal-title":"Mult Scler Relat Disord"},{"key":"hep41926-bib-0014-20241115","doi-asserted-by":"crossref","first-page":"1394","DOI":"10.3390\/v12121394","article-title":"IMU\u2010838, a developmental DHODH inhibitor in phase II for autoimmune disease, shows anti\u2010SARS\u2010CoV\u20102 and broad\u2010spectrum antiviral efficacy in 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