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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT00875160
Other study ID # GAU-CL-104
Secondary ID
Status Terminated
Phase Phase 1
First received April 2, 2009
Last updated August 17, 2010
Start date April 2009
Est. completion date July 2009

Study information

Verified date August 2010
Source Amicus Therapeutics
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug AdministrationUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Terminated
Enrollment 1
Est. completion date July 2009
Est. primary completion date July 2009
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 65 Years
Eligibility 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
AT2101
225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)

Locations

Country Name City State
United Kingdom Guy's Drug Research Unit London
United States Comprehensive Phase One Miramar Florida

Sponsors (1)

Lead Sponsor Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease Multiple times for up to three days following Day 1 and Day 10 No
Secondary To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease Daily Yes
Secondary To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease. Days 1, 5 & 7 (optional), 10 and 17 No
See also
  Status Clinical Trial Phase
Completed NCT00813865 - A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients Phase 2
Completed NCT00433147 - A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy Phase 2
Completed NCT00446550 - A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease Phase 2
Terminated NCT04836377 - A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Withdrawn NCT03021941 - Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease Phase 4