Ignite Creation Date:
2024-05-05 @ 5:23 PM
Last Modification Date:
2024-10-26 @ 9:31 AM
Study NCT ID:
NCT00446550
Status:
COMPLETED
Last Update Posted:
2018-08-15
First Post:
2007-03-09
Brief Title:
A Study of Oral AT2101 Afegostat Tartrate in Treatment-naive Patients With Gaucher Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
A Randomized Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher Disease
Status:
COMPLETED
Status Verified Date:
2018-07
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 evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy ERT or substrate reduction therapy SRT
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 21-day screening period a 24-week treatment period and follow-up visit Day 183 end-of-study Participants were randomized in a 11 ratio to 1 of 2 treatment regimens for afegostat tartrate 3 days on treatment4 days off or 7 days on treatment7 days off
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