20 December 2011 - Amicus Therapeutics (NASDAQ:FOLD), a US-based biopharmaceutical company focused on small molecule drugs for rare diseases, said on Monday that it had completed enrolment in the first Phase III study of migalastat HCl for Fabry disease.

According to Amicus and its partner in the study, GlaxoSmithKline (LON:GSK), results of the trial, called Study 011, are expected in the third quarter of 2012.

A total 67 patients were enrolled in the trial, more than the original target of 60.

As of December 2011, 21 of 23 patients who have completed the six-month treatment and six-month follow-up periods are currently enrolled in the ongoing Phase III extension study.

The primary efficacy endpoint for the trial is a change in interstitial capillary globotriaosylceramide (GL-3) as measured by kidney biopsy. Patients with a reduction of GL-3 deposits per capillary of at least 50% at six months will be considered responders. Secondary endpoints for the trial include safety and tolerability, urine GL-3, renal function, and quality of life (QOL).