A Randomized, 12-month, placebo-controlled, pivotal, cross-over study of migalastat in treatment naïve Fabry patients who have an amenable mutation and GI symptoms.
Phase of Trial: Phase III
Latest Information Update: 11 Jul 2017
At a glance
- Drugs Migalastat (Primary)
- Indications Fabry's disease
- Focus Registrational; Therapeutic Use
- 11 Jul 2017 According to an Amicus Therapeutics media release, this additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission.
- 06 Dec 2016 New trial record
- 28 Nov 2016 According to an Amicus Therapeutics media release, company is working with FDA to finalize the protocol of this trial and plans to initiate enrollment in 2017, with data expected in 2019. This trial will generate additional data which will support regulatory approval of migalastat for Fabry disease in the USA.