title: Inhaled alpha1-proteinase inhibitor therapy in patients with cystic fibrosis.
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.
authors: University of Alabama at Birmingham, Birmingham, AL, United States.
authors: Amit
authors: A
authors: Gaggar
authors: Clinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States.
authors: Junliang
authors: J
authors: Chen
authors: Division of Pediatric Pulmonology, Case Western Reserve, University School of Medicine and Rainbow Babies and Children's Hospital, Cleveland, OH, United States.
authors: James F
authors: JF
authors: Chmiel
authors: Harvard Medical School, Boston, MA, United States.
authors: Henry L
authors: HL
authors: Dorkin
authors: Medical University of South Carolina, Charleston, SC, United States.
authors: Patrick A
authors: PA
authors: Flume
authors: Clinical Development, Bioscience Industrial Group, Grifols Inc., Research Triangle Park, NC, United States. Electronic address: Rhonda.Griffin@grifols.com.
authors: Rhonda
authors: R
authors: Griffin
authors: National Jewish Health, Denver, CO, United States.
authors: David
authors: D
authors: Nichols
authors: University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC, United States.
authors: Scott H
authors: SH
authors: Donaldson
mesh_terms: D000280:Administration, Inhalation; D000328:Adult; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000378:metabolism; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D019272:Leukocyte Elastase / Q000097:blood; D008297:Male; D015842:Serine Proteinase Inhibitors / Q000008:administration & dosage / Q000493:pharmacokinetics; D013183:Sputum / Q000378:metabolism; D013997:Time Factors; D016896:Treatment Outcome; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage* / Q000493:pharmacokinetics
chemical_list: D015842:Serine Proteinase Inhibitors; D000515:alpha 1-Antitrypsin; D019272:Leukocyte Elastase
keywords: Alpha-1 HC; Alpha-1 antitrypsin; Cystic fibrosis; Prolastin