Conditions: Fabry Disease
Keywords: Amicus Therapeutics AT1001 Galafold Migalastat Substrate
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 9
Healthy volunteers: no

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

Detailed description:
This was a Phase 2, open-label study in female participants with Fabry disease. The study consisted of a 4-week screening period, during which participants' galactosidase (GLA) genotype was assessed for α-galactosidase A (α-Gal A) activity in response to migalastat. 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 once every other day (QOD) for 12 weeks during the treatment period and the optional 36-week extension period for a total treatment duration of up to 48 weeks. Participants were stratified by α-Gal A enzyme activity (high \>40%, and low ≤40%) then randomly assigned to receive migalastat at 1 of 3 specified dose levels (50, 150, or 250 milligrams \[mg\]).

Arms:
arm 1: Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period. arm 2: Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period. arm 3: Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.

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

Protocol document: