Ignite Creation Date:
2024-05-05 @ 5:21 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00433147
Status:
COMPLETED
Last Update Posted:
2018-09-25
First Post:
2007-02-07
Brief Title:
A Study of AT2101 Afegostat Tartrate in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
A Randomized Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Status:
COMPLETED
Status Verified Date:
2018-08
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:
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy
Detailed Description:
This was a Phase 2 open-label study in participants with Gaucher disease a lysosomal storage disorder Afegostat tartrate also known as AT2101 or isofagomine tartrate is designed to act as a pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase GCase and helping it fold correctly intended to restore GCase activity The study consisted of a 14-day screening period a 28-day treatment period and a 7-day wash-out period Participants received 1 of 4 dosing regimens for afegostat tartrate
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