[removed]Grifols[/removed], a health care company based in Barcelona, Spain, announced that the first patient has been enrolled and dosed in a phase II study evaluating the safety and tolerability of Alpha-1 HC aerosol in patients with cystic fibrosis (CF).
The multicenter phase II trial is a 3-week, dose-escalation, randomized, double-blind, placebo-controlled study to assess the safety and tolerability of 100 mg and 200 mg of Alpha-1 HC aerosol, an inhaled formulation of Grifols’ alpha1-proteinase inhibitor (human), administered once daily. The trial will enroll 30 patients at six sites across the United States. Study sites include the University of Alabama at Birmingham; Case Western Reserve Medical Center and Rainbow Babies and Children’s Hospital; Children’s Hospital Boston; Medical University of South Carolina; National Jewish Health, Denver; and the University of North Carolina at Chapel Hill.
On April 6, 2012, Grifols received orphan drug designation from the U.S. Food and Drug Administration (FDA) to develop Alpha-1 HC aerosol as a treatment for cystic fibrosis. Grifols also received a positive opinion on orphan drug designation for Alpha-1 HC aerosol as a treatment for cystic fibrosis from the European Medicines Agency on September 12, 2012.