Marc Dunoyer, Global Head of GSK Rare Diseases added, "GSK and Amicus are committed to advancing migalastat HCl as a monotherapy in Fabry patients with amenable mutations. While these 6-month data are encouraging, there is additional work to be done. We continue to analyze the 6-month results and look forward to receiving the 12-month results from this study. In addition the results of Study 012, our second Phase 3 Fabry monotherapy study, will add to the totality of our data and give us a more complete picture of the clinical effect of migalastat HCl. This study, an 18-month comparison of migalastat to ERT, with iohexol GFR as the primary endpoint, is fully recruited and due to report in 2014."
The 6-month primary treatment period in Study 011 was completed in June 2012 in 63 out of 67 randomized patients. All 63 of these patients entered the 6-month open-label follow-up period in Study 011, to continue to receive migalastat HCl or to switch from placebo to migalastat HCl. In December 2012, a total of 59 patients completed this treatment period and received an additional kidney biopsy at month 12. In addition, 57 out of 59 patients who completed Study 011 continue to receive migalastat HCl in both ongoing open-label extension studies. The results from the 6-12 month period of study 011 are expected in the first half of 2013 and will include 12-month data in the migalastat HCl group and 6-month data in the placebo crossover group.
A second Phase 3 global registration study ( Study 012) is also underway to compare open-label migalastat HCl to enzyme replacement therapy (ERT) to primarily support global registration. Study 012 (The ATTRACT, or FAB-AT1001-012 Study) is a randomized, open-label 18-month Phase 3 study investigating the safety and efficacy of oral migalastat HCl 150 mg QOD compared to standard-of-care infused therapy using ERTs (Fabrazyme® and Replagal®). This study achieved final enrollment of 60 total patients in December 2012.
Study 011 DesignStudy 011 - also referred to as FACETS - is one of two ongoing Phase 3 studies of migalastat HCl monotherapy being conducted by Amicus and GlaxoSmithKline (GSK). This study was designed based on feedback from the U.S. Food and Drug Administration (FDA), and is primarily intended to support U.S. registration. Study 011 randomized 67 patients (24 males and 43 females) diagnosed with Fabry disease who had genetic mutations amenable to chaperone monotherapy in a cell-based assay. For the 6-month, double-blind primary treatment period patients were randomized to migalastat HCl 150 mg or placebo on an every-other-day (QOD) oral dosing schedule. During a 6-month open-label follow up period, patients continued treatment with migalastat HCl or switched from placebo to migalastat HCl.
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