Fabry Disease Clinical Trial
Official title:
A Phase 2, Open-Label, Multicenter, 12-Week Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AT1001 in Patients With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
This was a Phase 2, open-label study in male participants with Fabry disease. All
participants who met initial eligibility criteria underwent a 28-day screening period,
including a 14-day run-in with migalastat (150 milligrams [mg] migalastat once a day [QD]
from Days -28 to -15) to assess eligibility for entering the treatment period of the study.
Participants who entered the treatment period were required to have α-galactosidase A (α-Gal
A) activity responsive to migalastat.
Fifteen participants received at least 1 dose of study drug, however, 6 of these participants
did not demonstrate α-Gal A activity responsive to migalastat and were thus screen failures
(these participants are hereafter referred to as "dosed screen failures") due to not meeting
all inclusion criteria for treatment. Therefore, 9 participants were enrolled into the
treatment period (these participants are hereafter referred to as "eligible-enrolled").
Eligible-enrolled participants (those who satisfied the criteria for inclusion in the study)
received escalating doses of migalastat twice a day (BID) for 6 weeks (Days 1 to 42),
followed by 6 weeks at 1 dose level BID (Days 43 to 84) during the treatment period.
Participants could then opt to participate in the extension period. The study consisted of 2
optional extension periods, the first through Week 48 and the second through Week 96. For
participants who did not continue into the optional treatment extension, the study included a
2-week follow-up period.
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