Ignite Creation Date:
2024-05-05 @ 4:44 PM
Last Modification Date:
2024-10-26 @ 9:23 AM
Study NCT ID:
NCT00304512
Status:
COMPLETED
Last Update Posted:
2018-10-03
First Post:
2006-03-17
Brief Title:
A 12-Week Safety and Pharmacodynamic Study of AT1001 Migalastat Hydrochloride in Female Participants With Fabry Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2018-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Study to evaluate the safety tolerability and pharmacodynamics of migalastat hydrochloride HCl migalastat in participants with Fabry disease
Detailed Description:
This was a Phase 2 open-label study in female participants with Fabry disease The study consisted of a 4-week screening period during which participants galactosidase GLA genotype was assessed for α-galactosidase A α-Gal A activity in response to migalastat Participants were required to have α-Gal A activity responsive to migalastat The study consisted of a 12-week treatment period followed by an optional 36-week extension period Participants received migalastat once every other day QOD for 12 weeks during the treatment period and the optional 36-week extension period for a total treatment duration of up to 48 weeks Participants were stratified by α-Gal A enzyme activity high 40 and low 40 then randomly assigned to receive migalastat at 1 of 3 specified dose levels 50 150 or 250 milligrams mg
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None