A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
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|ClinicalTrials.gov Identifier: NCT00875160|
Recruitment Status : Terminated (Subject Recruitment)
First Posted : April 3, 2009
Last Update Posted : August 19, 2010
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.
|Condition or disease||Intervention/treatment||Phase|
|Type 1 Gaucher Disease||Drug: AT2101||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||April 2009|
|Primary Completion Date :||July 2009|
|Study Completion Date :||July 2009|
225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)
- To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease [ Time Frame: Multiple times for up to three days following Day 1 and Day 10 ]
- To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease [ Time Frame: Daily ]
- To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease. [ Time Frame: Days 1, 5 & 7 (optional), 10 and 17 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00875160
|United States, Florida|
|Comprehensive Phase One|
|Miramar, Florida, United States, 33025|
|Guy's Drug Research Unit|
|London, United Kingdom, SE1 1YR|
|Study Director:||Eugene Schneider, MD||Amicus Therapeutics|