An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants
Phase of Trial: Phase III
Latest Information Update: 13 Dec 2018
At a glance
- Drugs Migalastat (Primary)
- Indications Fabry's disease
- Focus Adverse reactions; Pharmacokinetics; Registrational; Therapeutic Use
- Sponsors Amicus Therapeutics
- 14 Nov 2018 Planned number of patients changed from 10 to 20.
- 14 Nov 2018 Planned End Date changed from 1 May 2020 to 1 Sep 2020.
- 14 Nov 2018 Status changed from not yet recruiting to recruiting.