{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T15:25:06Z","timestamp":1778599506249,"version":"3.51.4"},"reference-count":34,"publisher":"European Respiratory Society (ERS)","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Eur Respir J"],"accepted":{"date-parts":[[2001,9,19]]},"published-print":{"date-parts":[[2002,2]]},"abstract":"<jats:p>The aim of the present studies was to investigate the tolerability and activity of a novel mucolytic drug, Nacystelyn (NAL), for the treatment of cystic fibrosis (CF) lung disease.<\/jats:p><jats:p>In study 1, involving 10 CF patients, the main objective was to determine the tolerability and potential efficacy of a range of single doses of NAL in comparison to a placebo, in order to establish an optimal dose for further testing. On five consecutive scheduled treatment days, patients inhaled either from two (4\u2005mg) to eight puffs (16\u2005mg) of a single dose of NAL from the range, administered in an open-label fashion, or 12 puffs of active NAL (24\u2005mg)<jats:italic>versus<\/jats:italic>12 puffs of placebo, administered in a randomized double-blind fashion. Pulmonary function data were unaffected and clinically-adverse effects were limited to wheezing in some patients that inhaled 12 puffs of either placebo or active drug. Subsequent rheological analysis of their sputum showed a dose-dependent decrease in sputum viscoelasticity, accompanied by a decrease in sputum solids content and an increase in chloride and sodium concentrations.<\/jats:p><jats:p>In study 2, involving 12 CF patients, the clinical safety and mucolytic activity of a single dose of NAL was monitored over 24\u2005h. On different scheduled treatment days, 7\u2005days apart, patients inhaled a single dose of 12 puffs of active NAL (24\u2005mg) or 12 puffs of placebo drug in a randomized, double-blind sequence, with sputum samples taken at intervals before and after inhalation. Mucus rigidity decreased following NAL inhalation, with the maximum effect observed at 4\u2005h; the 1-, 2- and 4-h NAL rheology results were significantly different from placebo. No adverse effects were observed.<\/jats:p><jats:p>The drug was well tolerated in both studies. Sputum results were predictive of improved clearability by ciliary and cough transport mechanisms.<\/jats:p>","DOI":"10.1183\/09031936.02.00025802","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T12:36:48Z","timestamp":1027773408000},"page":"294-302","source":"Crossref","is-referenced-by-count":49,"title":["Dose-finding and 24-h monitoring for efficacy and safety of aerosolized Nacystelyn in cystic fibrosis"],"prefix":"10.1183","volume":"19","author":[{"given":"E.M.","family":"App","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"D.","family":"Baran","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"I.","family":"Dab","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"Malfroot","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"Coffiner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"F.","family":"Vanderbist","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"King","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"81","published-online":{"date-parts":[[2002,2,1]]},"reference":[{"key":"2024101809053692000_19.2.294.1","unstructured":"National Institutes of Health (NIH)\/National Heart, Lung Blood Institute. 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Advanced Technology Corporation (ATC) Projects D-NAL-28D-96, D-NAL-182D-96, 1997 (internal report, University of Liege, Liege, Belgium)."},{"key":"2024101809053692000_19.2.294.30","doi-asserted-by":"crossref","first-page":"707","DOI":"10.1164\/ajrccm\/137.3.707","volume":"137","year":"1988","journal-title":"Am Rev Respir Dis"},{"key":"2024101809053692000_19.2.294.31","doi-asserted-by":"crossref","first-page":"1107","DOI":"10.1164\/ajrccm\/141.5_Pt_1.1107","volume":"141","year":"1990","journal-title":"Am Rev Respir Dis"},{"key":"2024101809053692000_19.2.294.32","doi-asserted-by":"crossref","first-page":"605","DOI":"10.1164\/ajrccm\/141.3.605","volume":"141","year":"1990","journal-title":"Am Rev Respir Dis"},{"key":"2024101809053692000_19.2.294.33","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1056\/NEJM199004263221704","volume":"322","year":"1990","journal-title":"N Engl J Med"},{"key":"2024101809053692000_19.2.294.34","unstructured":"Laboratoires SMB. 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