Conditions: Fabry Disease
Keywords: Amicus Therapeutics AT1001 Galafold Migalastat Substrate
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 4
Healthy volunteers: no

Brief summary:
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.

Detailed description:
This was a Phase 2, open-label study in male participants with Fabry disease. The study consisted of a 4-week screening period during which participants' genotype was assessed for α-galactosidase A (α-Gal A) activity in response to migalastat via an in vitro assay. Participants were required to have α-Gal A activity responsive to migalastat. The study consisted of a 12-week treatment period, followed by an optional 36-week extension period. Participants received migalastat 150 milligrams (mg) once every other day (QOD) for 12 weeks during the treatment period. Participants could then opt to participate in the extension period.

Arms:
arm 1: Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week extension period.

Interventions: intervention 1: migalastat HCl
Intervention descriptions: intervention 1: None

Protocol document: