Ignite Creation Date:
2025-12-25 @ 2:16 AM
Ignite Modification Date:
2025-12-26 @ 12:46 AM
Study NCT ID:
NCT00875160
Status:
TERMINATED
Last Update Posted:
2010-08-19
First Post:
2009-04-02
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
Sponsor:
Amicus Therapeutics
Organization:
Study Overview
Official Title:
An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease
Status:
TERMINATED
Status Verified Date:
2010-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Subject Recruitment
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days).
The study consists of a screening period (\~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.
The study consists of a screening period (\~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.
Detailed Description:
While on the study, subjects will be required to refrain from consuming the following foods and beverages:
* Caffeine: 24 hours before admission and throughout the in-patient stay;
* Alcohol: 48 hours before admission and throughout the in-patient stay. A maximum of 2 units/day will be allowed during the out-patient part of the study (1 unit of alcohol = wind (125mL)=spirits(25mL)=beer (284mL);
* Vitamins: throughout the in-patient periods.
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
* Caffeine: 24 hours before admission and throughout the in-patient stay;
* Alcohol: 48 hours before admission and throughout the in-patient stay. A maximum of 2 units/day will be allowed during the out-patient part of the study (1 unit of alcohol = wind (125mL)=spirits(25mL)=beer (284mL);
* Vitamins: throughout the in-patient periods.
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
Study Oversight
Has Oversight DMC:
False
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?: