A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
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|ClinicalTrials.gov Identifier: NCT00304512|
Recruitment Status : Completed
First Posted : March 20, 2006
Results First Posted : September 7, 2018
Last Update Posted : October 3, 2018
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat HCl||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease|
|Actual Study Start Date :||September 7, 2006|
|Actual Primary Completion Date :||May 9, 2008|
|Actual Study Completion Date :||May 9, 2008|
Experimental: Migalastat Low Dose 50 mg
Migalastat 50 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
Drug: migalastat HCl
Experimental: Migalastat Middle Dose 150 mg
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
Drug: migalastat HCl
Experimental: Migalastat High Dose 250 mg
Migalastat 250 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week treatment extension period.
Drug: migalastat HCl
- Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Day 1 (after dosing) through Week 48 ]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 48 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 hours (postdose) ]The AUC to the last measurable concentration (AUC0-t) was evaluated in plasma following a single oral dose of migalastat on Day 1 and following multiple oral doses on Day 14 (2 weeks) and Day 84 (12 weeks). All samples were collected on each dosing day.
- α-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48 [ Time Frame: Baseline, Week 12 (end of treatment period), Week 48 (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): NCT00304512
|United States, Georgia|
|Decatur, Georgia, United States, 30033|
|Parkville, Victoria, Australia, 3050|
|Porto Alegre, Brazil, RS, 90035-903|
|Montréal, Canada, H4J 1C5|
|Paris, France, 75015|
|Salford, United Kingdom, M6 8HD|
|Study Director:||Medical Monitor, Clinical Research||Amicus Therapeutics|