A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease
|ClinicalTrials.gov Identifier: NCT00283959|
Recruitment Status : Completed
First Posted : January 31, 2006
Last Update Posted : April 15, 2015
Information provided by (Responsible Party):
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Completed|
|Actual Primary Completion Date :||May 2008|
|Actual Study Completion Date :||May 2008|
|Certification/Extension First Submitted :||August 17, 2010|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.
Germain DP, Giugliani R, Hughes DA, Mehta A, Nicholls K, Barisoni L, Jennette CJ, Bragat A, Castelli J, Sitaraman S, Lockhart DJ, Boudes PF. Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis. 2012 Nov 24;7:91. doi: 10.1186/1750-1172-7-91.