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This phase 2, multi-center, open-label trial evaluated the efficacy and safety of Camostat Mesylate (CM), a serine protease inhibitor, as adjunctive therapy for PLE. Nineteen patients aged 4\u00a0years and older with PLE after the Fontan operation were enrolled. CM was administered for six months in addition to their individualized conventional treatments. Assessments were made at 1, 3, and 6\u00a0months of CM administration, and at one month after CM discontinuation. Outcomes evaluated were the changes in serum albumin level, stool alpha-1 antitrypsin, and clinical symptoms such as, diarrhea, edema, weight change, and ascites. Of the 19 patients enrolled, 4 voluntarily withdrew consent, and the data from the 15 patients who completed the study were analyzed. Their median age was 15.0\u00a0years (interquartile range, 12.0\u201321.5) and the median time between the Fontan surgery and PLE diagnosis was 2.4\u00a0years. Serum albumin levels increased from 2.2 to 2.5\u00a0g\/dL (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.183), while stool alpha-1 antitrypsin levels significantly decreased from 215.6 to 75.5\u00a0mg\/dL (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.016) over six months. Patients with baseline diarrhea showed notable improvements: serum albumin increased from 1.8 to 2.4\u00a0g\/dL (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.138) and stool alpha-1 antitrypsin decreased from 220.3 to 75.5\u00a0mg\/dL (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.075) over 6\u00a0months. No serious adverse events occurred. CM demonstrated significant reductions in gastrointestinal protein losses, particularly in patients with baseline diarrhea. Trial registration NCT05474664.\n                  <\/jats:p>","DOI":"10.1007\/s00246-025-03859-9","type":"journal-article","created":{"date-parts":[[2025,4,14]],"date-time":"2025-04-14T02:49:27Z","timestamp":1744598967000},"page":"932-940","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Phase 2 Open-label, Single-arm, Multi-center Clinical Trial to Evaluate the Efficacy and Safety of Camostat Mesylate in Patients with Protein-losing Enteropathy After Fontan Operation"],"prefix":"10.1007","volume":"47","clinical-trial-number":[{"clinical-trial-number":"nct05474664","registry":"10.18810\/clinical-trials-gov"}],"author":[{"given":"Woo Young","family":"Park","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gi Beom","family":"Kim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sang Yun","family":"Lee","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jae Suk","family":"Baek","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Soo Jin","family":"Kim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jowon","family":"Jung","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Myung Chul","family":"Hyun","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Young Tae","family":"Lim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"HyoungDoo","family":"Lee","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hoon","family":"Ko","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joowon","family":"Lee","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,4,14]]},"reference":[{"key":"3859_CR1","doi-asserted-by":"publisher","first-page":"1700","DOI":"10.1016\/j.jacc.2015.07.065","volume":"66","author":"KN Pundi","year":"2015","unstructured":"Pundi KN, Johnson JN, Dearani JA, Pundi KN, Li Z, Hinck CA, Dahl SH, Cannon BC, O\u2019Leary PW, Driscoll DJ (2015) 40-year follow-up after the Fontan operation: long-term outcomes of 1,052 patients. 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