RESEARCH TRIANGLE PARK, N.C. (Oct 8, 2012) — Grifols announced today 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. Cystic fibrosis is an inherited disease that causes progressive decline in lung function, including acute episodes of worsening respiratory symptoms and potentially fatal lung infections.
The multicenter trial will investigate two doses of Alpha-1 HC aerosol, an inhaled formulation of Grifols’ alpha1-proteinase inhibitor (human). Investigators will measure adverse events and other end points during the three-week study period. The randomized, double-blind, placebo-controlled trial will enroll 30 patients at six sites across the U.S.
"Our goal is to continually develop new therapies that will improve the quality of life and prognosis for individuals with cystic fibrosis, said Dr. Amit Gaggar, Associate Professor of Medicine at the University of Alabama, one of the study sites. “This study will offer insight into a novel therapeutic approach using an inhaled medicine that directly targets the affected lung tissue.”
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. Orphan drug designation is granted to encourage the development of treatments that prevent, diagnose or treat rare diseases that affect fewer than 200,000 people per year in the U.S. and fewer than 5 in 10,000 people in the EU.
Grifols leads the global market with its intravenous alpha1-proteinase inhibitor for the treatment of alpha1-antitrypsin deficiency, a rare disorder that can cause emphysema due to low circulating levels of the alpha1 protein in the lungs.
About the Study
The phase II trial is a three-week, dose-escalation, randomized, double-blind, placebo-controlled study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC aerosol administered once daily. 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, CO; and the University of North Carolina at Chapel Hill.