A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00283959|
Recruitment Status : Completed
First Posted : January 31, 2006
Results First Posted : September 7, 2018
Last Update Posted : October 31, 2018
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat HCl||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease|
|Actual Study Start Date :||June 27, 2006|
|Actual Primary Completion Date :||May 8, 2008|
|Actual Study Completion Date :||May 8, 2008|
Migalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week 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 (end of extension period) ]
TEAEs were defined as any adverse event with a 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.
- α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 12, And Week 48 [ Time Frame: Baseline, Week 12 (end of treatment period), Week 48 (end of extension period) ]
PBMCs were isolated from whole blood and lysed, and α-Gal A activity was measured by 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/hour [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 PBMCs are presented by individual participants. Values of "0" presented below represent α-Gal A activity levels that were below the lower limit of quantification.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00283959
|Porto Alegre, Brazil|
|Study Director:||Medical Monitor, Clinical Research||Amicus Therapeutics|