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

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

Detailed description:
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.

Arms:
arm 1: Migalastat was administered orally during the 12-week treatment period and then during the optional 2 treatment extension periods.

Treatment Period:

* Migalastat 25 mg BID for Weeks 1 and 2 (Day 1 through the morning dose on Day 14).
* Migalastat 100 mg BID for Weeks 3 and 4 (Day 15 through the morning dose on Day 28).
* Migalastat 250 mg BID for Weeks 5 and 6 (Day 29 through the morning dose on Day 42).
* Migalastat 25 mg BID for Weeks 6 to 12 (Days 43 to 84).

Extension Period:

* Migalastat 25 mg BID for Weeks 12 through 48.
* Migalastat 50 mg QD for Weeks 48 through 96.

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

Protocol document: