Year: 2015
Type: article
Study designs: Randomized Controlled Trial, Clinical Trial
Abstract: BACKGROUND: Inhaled alpha1-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 alpha1-PI formulation, in CF patients. METHODS: We performed a randomized, double-blind, placebo-controlled study and evaluated the safety of 100 or 200mg 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 alpha1-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.
Source: Journal of Cystic Fibrosis
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,370
Funders: Cystic Fibrosis Foundation, Gilead Sciences, Cystic Fibrosis Foundation Therapeutics, Children's Hospital Colorado, University of Washington, Case Western Reserve University, University of South Carolina, Seattle Children's Research Institute, Grifols, National Institutes of Health, Genentech, South Carolina Clinical and Translational Research Institute, Medical University of South Carolina, Korea National Institute of Health, National Institute of Diabetes and Digestive and Kidney Diseases, National Center for Advancing Translational Sciences
Awards: G3543393010, G3677118997, North Carolina Translational & Clinical Sciences Institute (NC TraCS), South Carolina Clinical & Translational Research Institute (SCTR), G454077636, North Carolina Translational and Clinical Science Institute (NC TraCS), G7136733553, G7565395483, UAB CF Research and Translation Core Center
OpenAlex ID: W1586387535