Journal
JOURNAL OF CYSTIC FIBROSIS
Volume 15, Issue 2, Pages 227-233Publisher
ELSEVIER
DOI: 10.1016/j.jcf.2015.07.009
Keywords
Cystic fibrosis; Alpha-1 HC; Alpha-1 antitrypsin; Prolastin
Categories
Funding
- Grifols Therapeutics Inc.
- Cystic Fibrosis Foundation [CFFT RETSCH14Y0]
- University of North Carolina Clinical and Translational Science Award [1UL1TR001111]
- South Carolina Clinical and Translational Research Institute/Medical University of South Carolina (National Institute of Health) [ULT TR000062]
- Grifols, Bioscience Industrial Group
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Background: Inhaled alphas-proteinase inhibitor (PI) is known to reduce neutrophil elastase burden in some patients with CF. This phase 2a study was designed to test inhaled Alpha-1 HC, a new aerosolized alphas-PI formulation, in CF patients. Methods: We performed a randomized, double-blind, placebo-controlled study and evaluated the safety of 100 or 200 mg of inhaled Alpha-1 HC once daily for 3 weeks in subjects with CF. Thirty adult subjects were randomized in a 2:1 ratio to receive Alpha-1 HC or placebo. Results: Drug delivery was confirmed by a dose-dependent increase in the sputum alphas-PI. Seven (20.0%) of the 35 adverse events in the 100-mg dose group, 3 (13.0%) of 23 in the 200-mg dose group, and 4 (14.3%) of 28 in the placebo group were drug-related in these subjects. One serious adverse event occurred in 1 subject within each group. Conclusions: Alpha-1 HC inhalation was safe and well tolerated. (C) 2015 The Authors. Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.
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