Back to resultsA Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
Primary Purpose
Type 1 Gaucher Disease
Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AT2101
Sponsored by
About this trial
This is an interventional treatment trial for Type 1 Gaucher Disease
Eligibility Criteria
Inclusion Criteria:
- 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 Criteria:
- 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.
Sites / Locations
- Comprehensive Phase One
- Guy's Drug Research Unit
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AT2101
Arm Description
Outcomes
Primary Outcome Measures
To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease
Secondary Outcome Measures
To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease
To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00875160
Brief Title
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
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 Type
Interventional
2. Study Status
Record Verification Date
August 2010
Overall Recruitment Status
Terminated
Why Stopped
Subject Recruitment
Study Start Date
April 2009 (undefined)
Primary Completion Date
July 2009 (Actual)
Study Completion Date
July 2009 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Amicus Therapeutics
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Gaucher Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AT2101
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
AT2101
Intervention Description
225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease
Time Frame
Daily
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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 Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Eugene Schneider, MD
Organizational Affiliation
Amicus Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Comprehensive Phase One
City
Miramar
State/Province
Florida
ZIP/Postal Code
33025
Country
United States
Facility Name
Guy's Drug Research Unit
City
London
ZIP/Postal Code
SE1 1YR
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
We'll reach out to this number within 24 hrs