Amit Gaggar1, Junliang Chen2, James F Chmiel3, Henry L Dorkin4, Patrick A Flume5, Rhonda Griffin6, David Nichols7, Scott H Donaldson8. 1. University of Alabama at Birmingham, Birmingham, AL, United States. 2. Clinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States. 3. Division of Pediatric Pulmonology, Case Western Reserve, University School of Medicine and Rainbow Babies and Children's Hospital, Cleveland, OH, United States. 4. Harvard Medical School, Boston, MA, United States. 5. Medical University of South Carolina, Charleston, SC, United States. 6. Clinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States. Electronic address: Rhonda.Griffin@grifols.com. 7. National Jewish Health, Denver, CO, United States. 8. University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC, United States.
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.
RCT Entities:
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 CFpatients. 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.
Authors: Gillian A Kelly-Robinson; James A Reihill; Fionnuala T Lundy; Lorcan P McGarvey; John C Lockhart; Gary J Litherland; Keith D Thornbury; S Lorraine Martin Journal: Int J Mol Sci Date: 2021-06-14 Impact factor: 5.923
Authors: Karina A Serban; Daniela N Petrusca; Andrew Mikosz; Christophe Poirier; Angelia D Lockett; Lauren Saint; Matthew J Justice; Homer L Twigg; Michael A Campos; Irina Petrache Journal: PLoS One Date: 2017-04-27 Impact factor: 3.240