Amicus Therapeutics announced it has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal (migalastat hydrochloride) for the treatment of Fabry disease. Amicus has begun submitting the Phase 3 protocol to investigational sites worldwide and expects to begin the dosing of subjects in the second half of 2009. The trial will consist of a six-month double-blind, randomized, placebo-controlled treatment stage and will enroll approximately 60 subjects who are naive to enzyme replacement therapy (ERT) or who have not received ERT for at least six months prior to the start of treatment with Amigal. The primary endpoint will be the change in the amount of kidney interstitial capillary GL-3 as measured in kidney biopsies using histology. Secondary endpoints will include safety and tolerability, kidney GL-3 as measured in urine, and an assessment of renal function (including glomerular filtration rate [GFR] and 24-hour urine protein).
Amigal is being investigated to treat Fabry disease, a lysosomal storage disorder caused by inherited genetic mutations in the GLA gene. Amigal is designed to selectively bind to and stabilize the enzyme alpha-galactosidase A (a-Gal A), which facilitates proper trafficking of the enzyme to the lysosomes, where it is needed to break down GL-3.
For more information call (609) 662-2000 or visit www.amicustherapeutics.com.