A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
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|ClinicalTrials.gov Identifier: NCT00214500|
Recruitment Status : Completed
First Posted : September 22, 2005
Results First Posted : September 7, 2018
Last Update Posted : October 30, 2018
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat HCl||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||January 2, 2006|
|Actual Primary Completion Date :||January 29, 2008|
|Actual Study Completion Date :||January 29, 2008|
Migalastat was administered orally during the 12-week treatment period and then during the optional 2 treatment extension periods.
Drug: migalastat HCl
- Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Day 1 (after dosing) through Week 96 ]TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 96 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
- PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat [ Time Frame: 0 (predose), 0.5, 1, 2, 3, 4, 5, 6, 8, and 10 hr (postdose) ]The AUC from time zero to 12 hours (hr) postdose (AUC0-12) was evaluated in plasma following a single dose of migalastat 25, 100, and 250 mg on Days 1, 15, and 29, respectively. In addition, AUC0-12 was assessed following multiple doses (14 days) of migalastat 25, 100, and 250 mg on Days 14, 28, and 42, respectively.
- α-Galactosidase A (α-Gal A) Activity In Leukocytes At Baseline, Week 12, And Week 96 [ Time Frame: Baseline, Week 12 (end of treatment period), Week 96 (end of extension period) ]Leukocytes were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole [nmol] 4-MU/hr) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00214500
|United States, California|
|Los Angeles, California, United States, 90048|
|United States, Georgia|
|Decatur, Georgia, United States, 30033|
|United States, Maryland|
|Bethesda, Maryland, United States, 20892|
|United States, New York|
|New York, New York, United States, 10016|
|United States, Texas|
|Houston, Texas, United States, 77030|
|Study Director:||Medical Monitor, Clinical Research||Amicus Therapeutics|