Date: 2012-10-09
Type of information: Treatment of the first patient
phase: 2
Announcement: enrollment and dosing of the first patient
Company: Grifols (Spain)
Product: Alpha-1 HC aerosol (inhaled formulation of human alpha1-proteinase inhibitor)
Action mechanism:
Disease: cystic fibrosis
Therapeutic area: Rare diseases - Genetic diseases
Country: USA
Trial
details: The randomized, double-blind, placebo-controlled trial will enroll 30 patients at six sites across the U.S. 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. Investigators will measure adverse events and other end points during the three-week study period.
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.
Latest
news: Grifols has announced that the first patient has been enrolled and dosed in a phase 2 study evaluating the safety and tolerability of Alpha-1 HC aerosol in patients with cystic fibrosis. The multicenter trial will investigate two doses of Alpha-1 HC aerosol, an inhaled formulation of Grifols’ alpha1-proteinase inhibitor (human).
On April 6, 2012, Grifols received orphan drug designation from the 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.
