Status:
TERMINATED
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
Lead Sponsor:
Amicus Therapeutics
Conditions:
Type 1 Gaucher Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
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 p...
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 hour...
Eligibility Criteria
Inclusion
- Have a confirmed diagnosis of type 1 Gaucher disease with a documented gene mutation;
- Clinically stable and on a stable dose of ERT for at least 2 years before study entry, with no changes to dose level or regimen in the last 6 months;
- Willing to stop ERT for the duration of the study;
- Male or female between the ages of 18 to 65 inclusive;
- All subjects of reproductive potential are required to practice an acceptable method of contraception;
- All subjects must have a body mass index of less than 30; and
- Provide written informed consent to participate in the study.
Exclusion
- A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study in the opinion of the Investigator;
- During the screening/wash out period, any clinically significant findings, based on physical exam, medical history review, lab assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the Investigator;
- Partial or total splenectomy;
- History of pulmonary hypertension or Gaucher-related lung disease;
- History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars;
- Screening or Day 1 (before dosing) 12-lead ECG demonstration QTc \>450 msec in males or \>470 in females;
- Pregnant or breast-feeding;
- Current/recent drug or alcohol abuse within the past 12 months;
- Treatment with any investigational product, including investigational forms of ERT, in the 90 days before study entry;
- Treatment in the previous 90 days with any drug known to have a well-defined potential for toxicity to a major organ;
- Presence or sequelae of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs; or
- Subject is otherwise unsuitable for the study in the opinion of the Investigator.
Key Trial Info
Start Date :
April 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2009
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT00875160
Start Date
April 1 2009
End Date
July 1 2009
Last Update
August 19 2010
Active Locations (2)
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1
Comprehensive Phase One
Miramar, Florida, United States, 33025
2
Guy's Drug Research Unit
London, United Kingdom, SE1 1YR